DE69232983D1 - Adeno-assozierter virus 2 basalvektoren - Google Patents

Adeno-assozierter virus 2 basalvektoren

Info

Publication number
DE69232983D1
DE69232983D1 DE69232983T DE69232983T DE69232983D1 DE 69232983 D1 DE69232983 D1 DE 69232983D1 DE 69232983 T DE69232983 T DE 69232983T DE 69232983 T DE69232983 T DE 69232983T DE 69232983 D1 DE69232983 D1 DE 69232983D1
Authority
DE
Germany
Prior art keywords
gene therapy
vectors
adeno
associated virus
basal
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Expired - Fee Related
Application number
DE69232983T
Other languages
English (en)
Other versions
DE69232983T2 (de
Inventor
Arun Srivastava
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Research Corp Technologies Inc
Original Assignee
Research Corp Technologies Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Research Corp Technologies Inc filed Critical Research Corp Technologies Inc
Publication of DE69232983D1 publication Critical patent/DE69232983D1/de
Application granted granted Critical
Publication of DE69232983T2 publication Critical patent/DE69232983T2/de
Anticipated expiration legal-status Critical
Expired - Fee Related legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/795—Porphyrin- or corrin-ring-containing peptides
    • C07K14/805—Haemoglobins; Myoglobins
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
DE69232983T 1991-11-08 1992-11-06 Adeno-assozierter virus 2 basalvektoren Expired - Fee Related DE69232983T2 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US07/789,917 US5252479A (en) 1991-11-08 1991-11-08 Safe vector for gene therapy
PCT/US1992/009769 WO1993009239A1 (en) 1991-11-08 1992-11-06 Adeno-associated virus-2 basal vectors

Publications (2)

Publication Number Publication Date
DE69232983D1 true DE69232983D1 (de) 2003-05-08
DE69232983T2 DE69232983T2 (de) 2003-11-13

Family

ID=25149101

Family Applications (1)

Application Number Title Priority Date Filing Date
DE69232983T Expired - Fee Related DE69232983T2 (de) 1991-11-08 1992-11-06 Adeno-assozierter virus 2 basalvektoren

Country Status (13)

Country Link
US (1) US5252479A (de)
EP (1) EP0566732B1 (de)
JP (2) JPH06504680A (de)
KR (1) KR930703453A (de)
AT (1) ATE236263T1 (de)
AU (1) AU657829B2 (de)
CA (1) CA2098483C (de)
DE (1) DE69232983T2 (de)
DK (1) DK0566732T3 (de)
ES (1) ES2192555T3 (de)
IL (1) IL103677A0 (de)
PT (1) PT101040B (de)
WO (1) WO1993009239A1 (de)

Families Citing this family (520)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6605712B1 (en) * 1990-12-20 2003-08-12 Arch Development Corporation Gene transcription and ionizing radiation: methods and compositions
US6410010B1 (en) * 1992-10-13 2002-06-25 Board Of Regents, The University Of Texas System Recombinant P53 adenovirus compositions
CA2108144A1 (en) 1991-03-06 1992-09-07 Jack A. Roth Methods and compositions for the selective inhibition of gene expression
US5747469A (en) 1991-03-06 1998-05-05 Board Of Regents, The University Of Texas System Methods and compositions comprising DNA damaging agents and p53
US6261834B1 (en) * 1991-11-08 2001-07-17 Research Corporation Technologies, Inc. Vector for gene therapy
US5587308A (en) * 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
AU676204B2 (en) 1992-09-18 1997-03-06 Canji, Inc. Gene therapy by retroviral vector with tumor suppressive gene
US5830461A (en) * 1992-11-25 1998-11-03 University Of Pittsburgh Of The Commonwealth System Of Higher Education Methods for promoting wound healing and treating transplant-associated vasculopathy
US5658565A (en) * 1994-06-24 1997-08-19 University Of Pittsburgh Of The Commonwealth System Of Higher Education Inducible nitric oxide synthase gene for treatment of disease
US6340674B1 (en) 1993-03-26 2002-01-22 Thomas Jefferson University Method of inhibiting the proliferation and causing the differentiation of cells with IGF-1 receptor antisense oligonucleotides
ES2196026T3 (es) * 1993-04-30 2003-12-16 Wellstat Biologics Corp Uso de ndv en la fabricacion de un medicamento para tratar el cancer.
US6686200B1 (en) * 1993-08-31 2004-02-03 Uab Research Foundation Methods and compositions for the large scale production of recombinant adeno-associated virus
WO1997003703A1 (en) * 1995-07-21 1997-02-06 Rhone-Poulenc Rorer Pharmaceuticals Inc. Adeno-associated viral liposomes and their use in transfecting dendritic cells to stimulate specific immunity
US5834441A (en) * 1993-09-13 1998-11-10 Rhone-Poulenc Rorer Pharmaceuticals Inc. Adeno-associated viral (AAV) liposomes and methods related thereto
US6652850B1 (en) 1993-09-13 2003-11-25 Aventis Pharmaceuticals Inc. Adeno-associated viral liposomes and their use in transfecting dendritic cells to stimulate specific immunity
US5693531A (en) * 1993-11-24 1997-12-02 The United States Of America As Represented By The Department Of Health And Human Services Vector systems for the generation of adeno-associated virus particles
FR2716893B1 (fr) * 1994-03-03 1996-04-12 Rhone Poulenc Rorer Sa Virus recombinants, leur préparation et leur utilisation thérapeutique.
IL113052A0 (en) 1994-03-23 1995-06-29 Rhone Poulenc Rorer Sa Recombinant viruses, their preparation and their use in gene therapy
DE4411402A1 (de) * 1994-03-31 1995-10-05 Juergen Schrader DNA-Expressionsvektoren zur Verwendung in der gentherapeutischen Behandlung von Gefäßerkrankungen
WO1995027494A1 (en) * 1994-04-11 1995-10-19 Sloan-Kettering Institute For Cancer Research DEFECTIVE HERPES AND DEFECTIVE ADENO-ASSOCIATED VIRUS VECTORS WITH p53 FOR THE TREATMENT OF CANCER
CA2187626C (en) * 1994-04-13 2009-11-03 Michael G. Kaplitt Aav-mediated delivery of dna to cells of the nervous system
WO1995032225A1 (en) * 1994-05-23 1995-11-30 The Salk Institute For Biological Studies Method for site-specific integration of nucleic acids and related products
US6814962B1 (en) * 1994-06-02 2004-11-09 Aventis Pharma S.A. Recombinant viruses and their use for treatment of atherosclerosis and other forms of coronary artery disease and method, reagent, and kit for evaluating susceptibility to same
US20020159979A1 (en) 1994-06-06 2002-10-31 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5658785A (en) * 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US6204059B1 (en) * 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
PT705903E (pt) 1994-08-12 2001-11-30 Myriad Genetics Inc Mutacoes no gene de susceptibilidade para o cancro da mama e do ovario ligado a 17q
ATE209660T1 (de) 1994-08-12 2001-12-15 Myriad Genetics Inc 17q-verbundenes ovarial- und brustkrebs empfindlichkeitsgen
US5871986A (en) 1994-09-23 1999-02-16 The General Hospital Corporation Use of a baculovirus to express and exogenous gene in a mammalian cell
US5652225A (en) * 1994-10-04 1997-07-29 St. Elizabeth's Medical Center Of Boston, Inc. Methods and products for nucleic acid delivery
US5856152A (en) * 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
CA2203809C (en) * 1994-10-28 2008-06-03 James M. Wilson Recombinant adenovirus and methods of use thereof
US5872005A (en) * 1994-11-03 1999-02-16 Cell Genesys Inc. Packaging cell lines for adeno-associated viral vectors
US5714170A (en) 1994-11-16 1998-02-03 Thomas Jefferson University Method of inducing resistance to tumor growth
US5998205A (en) 1994-11-28 1999-12-07 Genetic Therapy, Inc. Vectors for tissue-specific replication
US6638762B1 (en) 1994-11-28 2003-10-28 Genetic Therapy, Inc. Tissue-vectors specific replication and gene expression
FR2731710B1 (fr) * 1995-03-14 1997-04-30 Rhone Poulenc Rorer Sa Virus recombinants exprimant la lecithine cholesterol acyltransferase et utilisations en therapie genique
AU4954896A (en) * 1995-03-16 1996-10-02 Hisamitsu Pharmaceutical Co., Inc. Novel cell strains
FR2732357B1 (fr) * 1995-03-31 1997-04-30 Rhone Poulenc Rorer Sa Vecteurs viraux et utilisation pour le traitement des desordres hyperproliferatifs, notamment de la restenose
DE19513152A1 (de) 1995-04-07 1996-10-10 Bundesrep Deutschland Verwendung eines "Immundefizienzvirus-supprimierenden Lymphokins (ISL)" zur Hemmung der Virusvermehrung, insbesondere von Retroviren
US7745416B2 (en) * 1995-04-11 2010-06-29 The Regents Of The University Of California Method for in vivo regulation of cardiac muscle contractility
US6638264B1 (en) * 1995-05-16 2003-10-28 Biostratum Incorporation Perfusion apparatus and methods for pharmaceutical delivery
ATE226094T1 (de) * 1995-05-16 2002-11-15 Karl Tryggvason Verfahren zur arzneistoffabgabe mittels gentherapie
AU5741996A (en) * 1995-05-22 1996-12-11 Chiron Corporation Position-specific integration of vector constructs into eukaryotic genomes mediated by a chimeric integrase protein
US5756283A (en) * 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
US6281010B1 (en) 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
US6187757B1 (en) 1995-06-07 2001-02-13 Ariad Pharmaceuticals, Inc. Regulation of biological events using novel compounds
US5990388A (en) * 1995-06-07 1999-11-23 Research Corporation Technologies, Inc. Resistance to viruses and viroids in transgenic plants and animals expressing dsRNA-binding protein
US6110456A (en) * 1995-06-07 2000-08-29 Yale University Oral delivery or adeno-associated viral vectors
US20020068049A1 (en) * 1998-09-10 2002-06-06 Henderson Daniel R. Tissue specific adenoviral vectors
US5770720A (en) * 1995-08-30 1998-06-23 Barnes-Jewish Hospital Ubiquitin conjugating enzymes having transcriptional repressor activity
US5795872A (en) * 1995-09-19 1998-08-18 Pharmadigm, Inc. DNA construct for immunization
US6162796A (en) * 1995-09-27 2000-12-19 The Rockefeller University Method for transferring genes to the heart using AAV vectors
US6084085A (en) * 1995-11-14 2000-07-04 Thomas Jefferson University Inducing resistance to tumor growth with soluble IGF-1 receptor
CZ296810B6 (cs) * 1995-11-30 2006-06-14 Board Of Regents, The University Of Texas System Léciva pro lécbu nádoru
US5837492A (en) 1995-12-18 1998-11-17 Myriad Genetics, Inc. Chromosome 13-linked breast cancer susceptibility gene
WO1997022689A1 (en) 1995-12-18 1997-06-26 Myriad Genetics, Inc. Chromosome 13-linked breast cancer susceptibility gene
ES2325820T3 (es) 1995-12-22 2009-09-18 University Of Utah Research Foundation Gen del sindrome qt largo que codifica kvlqt1 y su asociacion con mink.
US5858351A (en) 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
US5962313A (en) * 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
US5846528A (en) * 1996-01-18 1998-12-08 Avigen, Inc. Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence
DE19608751B4 (de) * 1996-03-06 2006-05-18 Medigene Ag Verwendung eines Adeno-assoziierten Virus-Vektors zur Steigerung der Immunogenität von Zellen
US5952221A (en) * 1996-03-06 1999-09-14 Avigen, Inc. Adeno-associated virus vectors comprising a first and second nucleic acid sequence
GB9605124D0 (en) 1996-03-11 1996-05-08 Royal Free Hosp School Med Method of treating muscular disorders
US6723531B2 (en) 1996-04-05 2004-04-20 The Salk Institute For Biological Studies Method for modulating expression of exogenous genes in mammalian systems, and products related thereto
WO1997042820A1 (en) * 1996-05-16 1997-11-20 Duke University Tristetraprolin
US6534314B1 (en) 1996-06-14 2003-03-18 Massachusetts Institute Of Technology Methods and compositions for transforming cells
US5780447A (en) * 1996-06-14 1998-07-14 St. Jude Children's Research Hospital Recombinant adeno-associated viral vectors
US5928914A (en) * 1996-06-14 1999-07-27 Albert Einstein College Of Medicine Of Yeshiva University, A Division Of Yeshiva University Methods and compositions for transforming cells
EP0979101B1 (de) 1996-07-03 2010-10-27 Merial, Inc. Rekombinanter hunde-adenovirus 2 (cav2), welcher exogene dna enthält
JP2001500376A (ja) * 1996-09-06 2001-01-16 カイロン コーポレイション 組換えａａｖベクターを使用する治療分子の肝臓特異的送達のための方法および組成物
US5866552A (en) * 1996-09-06 1999-02-02 The Trustees Of The University Of Pennsylvania Method for expressing a gene in the absence of an immune response
US20020037867A1 (en) * 1999-02-26 2002-03-28 James M. Wilson Method for recombinant adeno-associated virus-directed gene therapy
WO1998012311A2 (en) 1996-09-11 1998-03-26 The General Hospital Corporation Use of a non-mammalian dna virus to express an exogenous gene in a mammalian cell
US6544523B1 (en) 1996-11-13 2003-04-08 Chiron Corporation Mutant forms of Fas ligand and uses thereof
WO1998024924A1 (en) * 1996-12-05 1998-06-11 Introgene B.V. Genetic modification of primate hemopoietic repopulating stem cells
US7008776B1 (en) 1996-12-06 2006-03-07 Aventis Pharmaceuticals Inc. Compositions and methods for effecting the levels of high density lipoprotein (HDL) cholesterol and apolipoprotein AI very low density lipoprotein (VLDL) cholesterol and low density lipoprotein (LDL) cholesterol
GB9701425D0 (en) 1997-01-24 1997-03-12 Bioinvent Int Ab A method for in vitro molecular evolution of protein function
US6218597B1 (en) 1997-04-03 2001-04-17 University Technology Corporation Transgenic model and treatment for heart disease
CN1177934C (zh) 1997-04-28 2004-12-01 阿文蒂斯药物股份有限公司 腺病毒介导的肿瘤内投送血管生成拮抗剂用于肿瘤的治疗
US6541036B1 (en) 1997-05-29 2003-04-01 Thomas Jefferson University Treatment of tumors with oligonucleotides directed to insulin-like growth factor-I receptors (IGF-IR)
US6156303A (en) * 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6635623B1 (en) 1997-06-13 2003-10-21 Baylor College Of Medicine Lipoproteins as nucleic acid vectors
US20050096288A1 (en) * 1997-06-13 2005-05-05 Aragene, Inc. Lipoproteins as nucleic acid vectors
GB9712512D0 (en) 1997-06-16 1997-08-20 Bioinvent Int Ab A method for in vitro molecular evolution of protein function
US6300488B1 (en) 1997-07-10 2001-10-09 The Salk Institute For Biological Studies Modified lepidopteran receptors and hybrid multifunctional proteins for use in transcription and regulation of transgene expression
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
EP1027451A1 (de) * 1997-10-08 2000-08-16 Advanced Research & Technology Institute Chimäres parvovirales rekombinantes vektorsystem, welches spezifisch für erythroide zelllinien ist
US6346415B1 (en) * 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
IL135578A0 (en) 1997-10-29 2001-05-20 Genzyme Corp Compositions and methods for treating lysosomal storage disease
BR9813930A (pt) 1997-11-06 2006-12-19 Chiron Spa antìgeno neisserial
ATE302267T1 (de) 1998-01-08 2005-09-15 Aventis Pharma Inc Transgenes kaninchen, dass ein funktionelles menschliches lipoprotein(a) exprimiert
US6780609B1 (en) 1998-10-23 2004-08-24 Genome Therapeutics Corporation High bone mass gene of 1.1q13.3
EP1047784B2 (de) 1998-01-14 2015-03-18 Novartis Vaccines and Diagnostics S.r.l. Antigene aus neisseria meningitidis
US6984635B1 (en) 1998-02-13 2006-01-10 Board Of Trustees Of The Leland Stanford Jr. University Dimerizing agents, their production and use
NZ532665A (en) 1998-05-01 2005-11-25 Inst Genomic Research Neisseria meningitidis antigens and compositions
US6225456B1 (en) 1998-05-07 2001-05-01 University Technololy Corporation Ras suppressor SUR-5
US6333318B1 (en) 1998-05-14 2001-12-25 The Salk Institute For Biological Studies Formulations useful for modulating expression of exogenous genes in mammalian systems, and products related thereto
US6506889B1 (en) 1998-05-19 2003-01-14 University Technology Corporation Ras suppressor SUR-8 and related compositions and methods
US7153655B2 (en) * 1998-06-16 2006-12-26 Alligator Bioscience Ab Method for in vitro molecular evolution of protein function involving the use of exonuclease enzyme and two populations of parent polynucleotide sequence
US6135976A (en) 1998-09-25 2000-10-24 Ekos Corporation Method, device and kit for performing gene therapy
US6743906B1 (en) 1998-10-02 2004-06-01 Board Of Regents, The University Of Texas PPP2R1B is a tumor suppressor
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US6200560B1 (en) * 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US7129043B1 (en) * 1998-10-22 2006-10-31 Duke University Methods of screening for risk of proliferative disease and methods for the treatment of proliferative disease
US6759237B1 (en) * 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
US6855549B1 (en) 1998-11-23 2005-02-15 The University Of Iowa Research Foundation Methods and compositions for increasing the infectivity of gene transfer vectors
US6441156B1 (en) * 1998-12-30 2002-08-27 The United States Of America As Represented By The Department Of Health And Human Services Calcium channel compositions and methods of use thereof
US6387368B1 (en) 1999-02-08 2002-05-14 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
US6495376B1 (en) 1999-02-18 2002-12-17 Beth Israel Deaconess Medical Center Methods and compositions for regulating protein-protein interactions
US7794703B1 (en) * 1999-02-19 2010-09-14 Hai Xing Chen Method for production and delivery of a protein in vivo
US20030215423A1 (en) * 1999-04-01 2003-11-20 Merck & Co., Inc. Gene therapy for obesity
US20080145347A1 (en) * 1999-04-29 2008-06-19 Steiner Mitchell S p-Hyde sequences in the Rat
NZ571167A (en) 1999-04-30 2010-05-28 Novartis Vaccines & Diagnostic Fragments from Neisseria protein ORF 953 and their use in medicaments and diagnostic reagents
GB9911683D0 (en) 1999-05-19 1999-07-21 Chiron Spa Antigenic peptides
US6165754A (en) * 1999-06-08 2000-12-26 Cornell Research Foundation, Inc. Method of expressing an exogenous nucleic acid
US7504253B2 (en) * 1999-06-11 2009-03-17 The Burnham Institute For Medical Research Nucleic acid encoding proteins involved in protein degradation, products and methods related thereof
GB9916529D0 (en) 1999-07-14 1999-09-15 Chiron Spa Antigenic peptides
CA2382045A1 (en) * 1999-08-20 2001-03-01 Board Of Regents, The University Of Texas System Hdac4 and hdac5 in the regulation of cardiac gene expression
US7057015B1 (en) 1999-10-20 2006-06-06 The Salk Institute For Biological Studies Hormone receptor functional dimers and methods of their use
EP2975127A1 (de) 1999-10-29 2016-01-20 GlaxoSmithKline Biologicals SA Neisseria antigenpeptide
AU3642601A (en) * 1999-11-05 2001-05-30 Avigen, Inc. Ecdysone-inducible adeno-associated virus expression vectors
WO2001036603A2 (en) * 1999-11-17 2001-05-25 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
JP2003516731A (ja) 1999-11-18 2003-05-20 カイロン コーポレイション ヒトｆｇｆ−２１遺伝子および遺伝子発現産物
US6241710B1 (en) 1999-12-20 2001-06-05 Tricardia Llc Hypodermic needle with weeping tip and method of use
EP2275129A3 (de) 2000-01-17 2013-11-06 Novartis Vaccines and Diagnostics S.r.l. Ergänzter Außenmembranvesikel-Impfstoff gegen Meningokokken
EP1854476A3 (de) 2000-02-09 2008-05-07 Bas Medical, Inc. Verwendung von Relaxin zur Behandlung von durch Vasokonstriktion bedingten Krankheiten
AU3988401A (en) * 2000-02-25 2001-09-03 Ludwig Inst Cancer Res Materials and methods involving hybrid vascular endothelial growth factor dnas and proteins
WO2001066595A2 (en) 2000-03-08 2001-09-13 Chiron Corporation Human fgf-23 gene and gene expression products
GB0018307D0 (en) 2000-07-26 2000-09-13 Aventis Pharm Prod Inc Polypeptides
EP1950297A2 (de) 2000-05-31 2008-07-30 Novartis Vaccines and Diagnostics, Inc. Zusammensetzungen und Verfahren zur Behandlung neoplastischer Erkrankungen mithilfe von Chemotherapie und Strahlungssensibilisatoren
US7198924B2 (en) 2000-12-11 2007-04-03 Invitrogen Corporation Methods and compositions for synthesis of nucleic acid molecules using multiple recognition sites
SG165981A1 (en) 2000-10-27 2010-11-29 Chiron Srl Nucleic acids and proteins from streptococcus groups a & b
US20030188326A1 (en) 2000-11-03 2003-10-02 Dana Farber Cancer Institute Methods and compositions for the diagnosis of cancer susceptibilities and defective DNA repair mechanisms and treatment thereof
US20030093819A1 (en) 2000-11-03 2003-05-15 D'andrea Alan D. Methods and compositions for the diagnosis of cancer susceptibilities and defective DNA repair mechanisms and treatment thereof
US6958213B2 (en) 2000-12-12 2005-10-25 Alligator Bioscience Ab Method for in vitro molecular evolution of protein function
US20020086292A1 (en) 2000-12-22 2002-07-04 Shigeaki Harayama Synthesis of hybrid polynucleotide molecules using single-stranded polynucleotide molecules
DE60144198D1 (de) 2000-12-28 2011-04-21 Wyeth Llc Rekombinantes schutzprotein aus streptococcus pneumoniae
US6613534B2 (en) 2001-03-20 2003-09-02 Wake Forest University Health Sciences MAP-2 as a determinant of metastatic potential
GB0107658D0 (en) 2001-03-27 2001-05-16 Chiron Spa Streptococcus pneumoniae
GB0107661D0 (en) 2001-03-27 2001-05-16 Chiron Spa Staphylococcus aureus
WO2002081639A2 (en) 2001-04-06 2002-10-17 Georgetown University Gene brcc2 and diagnostic and therapeutic uses thereof
US20040242523A1 (en) * 2003-03-06 2004-12-02 Ana-Farber Cancer Institue And The Univiersity Of Chicago Chemo-inducible cancer gene therapy
US8034791B2 (en) 2001-04-06 2011-10-11 The University Of Chicago Activation of Egr-1 promoter by DNA damaging chemotherapeutics
WO2002081641A2 (en) 2001-04-06 2002-10-17 Georgetown University Gene scc-112 and diagnostic and therapeutic uses thereof
AU2002258728A1 (en) 2001-04-06 2002-10-21 Georgetown University Gene brcc-3 and diagnostic and therapeutic uses thereof
EP1390075B1 (de) * 2001-04-06 2012-01-18 The University of Chicago Chemotherapeutische einleitung der egr-1-promoter-aktivität in gentherapie
WO2002097056A2 (en) * 2001-05-31 2002-12-05 The Rockefeller University Method for generating replication defective viral vectors that are helper free
AUPR546801A0 (en) 2001-06-05 2001-06-28 Commonwealth Scientific And Industrial Research Organisation Recombinant antibodies
JP2005500835A (ja) * 2001-06-29 2005-01-13 ノバルティス アクチエンゲゼルシャフト Ｐｅｒｖスクリーニング法およびその使用
MX339524B (es) 2001-10-11 2016-05-30 Wyeth Corp Composiciones inmunogenicas novedosas para la prevencion y tratamiento de enfermedad meningococica.
EP2335723A1 (de) 2001-12-12 2011-06-22 Novartis Vaccines and Diagnostics S.r.l. Immunisierung gegen Chlamydia trachomatis
CN100593544C (zh) 2002-03-15 2010-03-10 惠氏控股有限公司 酶活性减少的非典型流感嗜血杆菌的p4蛋白突变体
US7384738B2 (en) * 2002-03-28 2008-06-10 Bremel Robert D Retrovirus-based genomic screening
US7244565B2 (en) 2002-04-10 2007-07-17 Georgetown University Gene shinc-3 and diagnostic and therapeutic uses thereof
WO2003087367A2 (en) * 2002-04-18 2003-10-23 Lynkeus Biotech Gmbh Means and methods for the specific inhibition of genes in cells and tissue of the cns and/or eye
CA2485506C (en) * 2002-05-17 2012-02-28 Alligator Bioscience Ab A method for in vitro molecular evolution of protein function
WO2003100008A2 (en) 2002-05-24 2003-12-04 Schering Corporation Neutralizing human anti-igfr antibody
US7148342B2 (en) * 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis
US7785608B2 (en) 2002-08-30 2010-08-31 Wyeth Holdings Corporation Immunogenic compositions for the prevention and treatment of meningococcal disease
CN102304570B (zh) 2002-11-13 2015-01-21 托马斯杰斐逊大学 用于癌症诊断和治疗的组合物和方法
AU2003298816C1 (en) 2002-12-02 2010-12-16 Amgen Fremont, Inc. Antibodies directed to Tumor Necrosis Factor and uses thereof
US20050048041A1 (en) * 2003-01-13 2005-03-03 Rao Mahendra S. Persistent expression of candidate molecule in proliferating stem and progenitor cells for delivery of therapeutic products
AU2004205895B2 (en) 2003-01-16 2009-02-26 The Trustees Of The University Of Pennsylvania Compositions and methods for siRNA inhibition of ICAM-1
GB0308198D0 (en) 2003-04-09 2003-05-14 Chiron Srl ADP-ribosylating bacterial toxin
US20050028229A1 (en) * 2003-04-14 2005-02-03 Mitchell Weiss Alpha-hemoglobin stabilizing protein transgenic mouse and methods of use thereof
CN1816565A (zh) * 2003-05-21 2006-08-09 阿雷斯贸易股份有限公司 Tnf－样分泌性蛋白质
AU2004272114B2 (en) * 2003-09-12 2010-11-04 Vertex Pharmaceuticals Incorporated Animal model for protease activity and liver damage
US20060078558A1 (en) * 2003-11-12 2006-04-13 Whitsett Jeffrey A Diagnosis, prognosis and treatment of pulmonary diseases
EP2287341B1 (de) 2003-12-01 2013-02-13 Life Technologies Corporation Rekombinationsstellen enthaltende Nukleinsäuremoleküle und Verfahren zur Verwendung davon
WO2005067708A2 (en) 2004-01-14 2005-07-28 Daniolabs Limited Zebrafish model for autoimmune diseases
US7432057B2 (en) 2004-01-30 2008-10-07 Michigan State University Genetic test for PSE-susceptible turkeys
WO2005078139A2 (en) 2004-02-09 2005-08-25 Thomas Jefferson University DIAGNOSIS AND TREATMENT OF CANCERS WITH MicroRNA LOCATED IN OR NEAR CANCER-ASSOCIATED CHROMOSOMAL FEATURES
AU2005222384A1 (en) * 2004-03-05 2005-09-22 Vegenics Limited Growth factor binding constructs materials and methods
AU2005222902B2 (en) * 2004-03-12 2010-06-10 Alnylam Pharmaceuticals, Inc. iRNA agents targeting VEGF
US7582442B2 (en) 2004-03-16 2009-09-01 The Regents Of The University Of Michigan Methods and compositions for using aleveolar macrophage phospholipase A2
US7319015B2 (en) * 2004-03-16 2008-01-15 The Regents Of The University Of Michigan Methods and compositions for using alveolar macrophage phospholipase A2
CN104292321A (zh) 2004-03-29 2015-01-21 株式会社嘉尔药物 新的半乳凝素9 突变体蛋白质及其用途
US7604798B2 (en) 2004-07-15 2009-10-20 Northwestern University Methods and compositions for importing nucleic acids into cell nuclei
US20090233986A1 (en) * 2004-07-27 2009-09-17 Mount Sinai School Of Medicine Methods and compositions for using sax2
CA2577550C (en) * 2004-08-25 2012-10-16 The University Of Chicago Use of the combination comprising temozolomide and tnf-alpha for treating glioblastoma
FI20050753A (fi) 2004-09-03 2006-03-04 Licentia Oy Uudet peptidit
GB0423126D0 (en) * 2004-10-18 2004-11-17 Ares Trading Sa Protein
US8809287B2 (en) * 2004-11-15 2014-08-19 Icahn School Of Medicine At Mount Sinai Compositions and methods for altering Wnt autocrine signaling
GB0426960D0 (en) 2004-12-08 2005-01-12 Ares Trading Sa TGR-3 like protein receptor
WO2006101629A2 (en) * 2005-02-17 2006-09-28 Vertex Pharmaceuticals Incorporated SODIUM CHANNEL PROTEIN TYPE III α-SUBUNIT SPLICE VARIANT
GB0504767D0 (en) * 2005-03-08 2005-04-13 Ares Trading Sa Lipocalin protein
US20060216315A1 (en) * 2005-03-16 2006-09-28 Yoo Tai J Cockroach allergen gene expression and delivery systems and uses
WO2006102643A2 (en) * 2005-03-24 2006-09-28 Caritas St. Elizabeth Medical Center Boston, Inc. Stably transformed bone marrow-derived cells and uses thereof
KR20080025373A (ko) 2005-05-17 2008-03-20 아미쿠스 세라퓨틱스, 인코포레이티드 1-데옥시노지리마이신 및 유도체를 이용한 폼페병의 치료방법
US20090270479A1 (en) * 2005-07-12 2009-10-29 Antonio Giordano Genetic and Epigenetic Alterations In the Diagnosis and Treatment of Cancer
WO2007016643A2 (en) * 2005-08-01 2007-02-08 Mount Sinai School Of Medicine Of New York University A method for extending longevity using npc1l1 antagonists
BRPI0614366A2 (pt) 2005-08-11 2009-10-06 Arpi Matossian Rogers peptìdeo, anticorpo ou fragmento de anticorpo, anticorpo ou ligante funcionalmente equivalente, molécula de ácido nucleico, vetor, célula hospedeira, métodos para expressar um peptìdeo, um anticorpo ou fragmento de anticorpo, um anticorpo ou ligante equivalente e para tratar uma doença em um paciente, composição farmacêutica, composição de vacina, métodos de vacinação de um indivìduo contra uma doença ou um distúrbio e de diagnose de um indivìduo para a presença de anticorpos autoimunes, e, arranjo de peptìdeos
WO2007022287A2 (en) 2005-08-15 2007-02-22 Vegenics Limited Modified vegf and pdgf with improved angiogenic properties
AU2006291165B2 (en) 2005-09-12 2013-03-14 The Ohio State University Research Foundation Compositions and methods for the diagnosis and therapy of BCL2-associated cancers
US20080200408A1 (en) * 2005-09-30 2008-08-21 Mccormack Kenneth Deletion mutants of tetrodotoxin-resistant sodium channel alpha subunit
US7972813B2 (en) * 2005-09-30 2011-07-05 Vertex Pharmaceuticals Incorporated Tetrodotoxin-resistant sodium channel alpha subunit
GB2432366B (en) * 2005-11-19 2007-11-21 Alligator Bioscience Ab A method for in vitro molecular evolution of protein function
CA2524619A1 (en) * 2005-11-22 2007-05-22 Ottawa Health Research Institute Novel stem cells, nucleotide sequences and proteins therefrom
DE602005023550D1 (de) 2005-12-14 2010-10-21 Licentia Ltd Verwendungen eines neurotrophischen Faktors
MX2008008302A (es) * 2005-12-22 2009-01-21 Exegenics Inc Composiciones y metodos para regular el sistema de complemento.
EP1971690B1 (de) 2005-12-28 2018-10-17 Translational Therapeutics, Inc. Auf translationaler fehlfunktion beruhende therapeutika
CA2633754C (en) 2006-01-05 2013-06-18 The Ohio State University Research Foundation Microrna-based methods and compositions for the diagnosis and treatment of solid cancers
EP2591794A1 (de) 2006-01-05 2013-05-15 The Ohio State University Research Foundation Anomalien bei Mikro-RNA-Expressionen in endokrinen und azinösen Pankreastumoren
WO2007081720A2 (en) 2006-01-05 2007-07-19 The Ohio State University Research Foundation Microrna-based methods and compositions for the diagnosis, prognosis and treatment of lung cancer
US20090214496A1 (en) * 2006-01-30 2009-08-27 Licentia Ltd. Bmx/etk tyrosine kinase gene therapy materials and methods
US8229398B2 (en) * 2006-01-30 2012-07-24 Qualcomm Incorporated GSM authentication in a CDMA network
EP2522749A1 (de) 2006-03-02 2012-11-14 The Ohio State University Mikro-RNA-Expressionsprofil im Zusammenhang mit Bauchspeicheldrüsenkrebs
US8252547B2 (en) 2006-03-20 2012-08-28 Japan Science And Technology Agency In vitro control of protein synthesis in mammalian cells by IP3 receptor-binding protein
EP2371971B1 (de) 2006-03-20 2013-11-27 The Ohio State University Research Foundation Mikro-RNA-Fingerabdrücke während humaner Megakaryozytopoiese
NZ571568A (en) 2006-03-31 2010-11-26 Alnylam Pharmaceuticals Inc Double-stranded RNA molecule compositions and methods for inhibiting expression of Eg5 gene
JP2009532057A (ja) 2006-04-07 2009-09-10 ニューロ セラピューティクス エービー 神経細胞の生存および発生
WO2007127919A2 (en) * 2006-04-28 2007-11-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a gene from the jc virus
US7723564B2 (en) 2006-05-10 2010-05-25 Board Of Regents Of The University Of Nebraska Compositions and methods for modulation of KSR1 and KSR2 interactions
CN103614375A (zh) 2006-05-11 2014-03-05 阿尔尼拉姆医药品有限公司 抑制pcsk9基因表达的组合物和方法
ES2429225T3 (es) 2006-05-17 2013-11-13 The Ludwig Institute For Cancer Research Anticuerpos anti VEGF-B para tratamiento o profilaxis de diabetes de tipo II o síndrome metabólico
CA2652770A1 (en) 2006-05-19 2007-11-29 Alnylam Pharmaceuticals, Inc. Rnai modulation of aha and therapeutic uses thereof
WO2007137220A2 (en) 2006-05-22 2007-11-29 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of ikk-b gene
US8598333B2 (en) * 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
ATE522541T1 (de) 2006-06-09 2011-09-15 Novartis Ag Bakterielle adhäsine konformere
ES2390499T3 (es) * 2006-06-12 2012-11-13 Opko Pharmaceuticals, Llc Composiciones y métodos para la inhibición de la angiogénesis por sirna
EP2455493B1 (de) 2006-07-13 2014-01-08 The Ohio State University Research Foundation Verfahren und Zusammensetzungen auf Mikro-RNA-Basis zur Diagnose und Behandlung von Krankheiten im Zusammenhang mit dem Dickdarm
RU2500815C2 (ru) 2006-07-28 2013-12-10 Санофи-Авентис Композиция и способ лечения опухолей
US7872118B2 (en) * 2006-09-08 2011-01-18 Opko Ophthalmics, Llc siRNA and methods of manufacture
AU2007299629C1 (en) 2006-09-21 2012-05-10 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the HAMP gene
ES2388567T3 (es) 2006-10-19 2012-10-16 Csl Limited Anticuerpos anti-il-13r alfa 1 y usos de los mismos
WO2008060813A2 (en) 2006-10-19 2008-05-22 Merck & Co., Inc. High affinity antibody antagonists of interleukin-13 receptor alpha 1
US20080113915A1 (en) * 2006-11-09 2008-05-15 Duke University SAPAP3 knockout mouse and clinical modeling associated with the SAPAP3 gene
AR064642A1 (es) 2006-12-22 2009-04-15 Wyeth Corp Polinucleotido vector que lo comprende celula recombinante que comprende el vector polipeptido , anticuerpo , composicion que comprende el polinucleotido , vector , celula recombinante polipeptido o anticuerpo , uso de la composicion y metodo para preparar la composicion misma y preparar una composi
MX2009008413A (es) * 2007-02-06 2009-11-02 Tai June Yoo Tratamiento y prevencion de enfermedades neurodegenerativas usando terapia genica.
WO2008115387A2 (en) * 2007-03-15 2008-09-25 University Hospitals Of Cleveland Screening, diagnosing, treating and prognosis of pathophysiologic states by rna regulation
PE20090064A1 (es) 2007-03-26 2009-03-02 Novartis Ag Acido ribonucleico de doble cadena para inhibir la expresion del gen e6ap humano y composicion farmaceutica que lo comprende
AP3018A (en) 2007-03-29 2014-10-31 Alnylam Pharmaceuticals Inc Compositions and methods for inhibiting expressionof a gene from the ebola
US20100184823A1 (en) 2007-07-05 2010-07-22 Mark Aron Labow dsRNA For Treating Viral Infection
WO2009012263A2 (en) * 2007-07-18 2009-01-22 The Trustees Of Columbia University In The City Of New York Tissue-specific micrornas and compositions and uses thereof
US8367632B2 (en) 2007-07-31 2013-02-05 Ohio State University Research Foundation Methods for reverting methylation by targeting methyltransferases
EP2173908B1 (de) 2007-08-03 2016-01-06 The Ohio State University Research Foundation Ultrakonservierte bereiche zur kodierung von ncrnas
WO2009026487A1 (en) 2007-08-22 2009-02-26 The Ohio State University Research Foundation Methods and compositions for inducing deregulation of epha7 and erk phosphorylation in human acute leukemias
EP2775001B1 (de) 2007-09-06 2016-03-09 The Ohio State University Research Foundation MikroRNA-Signaturen in menschlichem Ovarialkarzinom
CA2702241A1 (en) 2007-10-11 2009-04-16 The Ohio State University Research Foundation Methods and compositions for the diagnosis and treatment of esophageal adenocarcinomas
FI20070808A0 (fi) 2007-10-25 2007-10-25 Mart Saarma GDNF:n silmukointivariantit ja niiden käytöt
EP2245159A2 (de) 2007-12-10 2010-11-03 Alnylam Pharmaceuticals Inc. Zusammensetzungen und verfahren zur hemmung der expression des faktor-vii-gens
US20090196854A1 (en) * 2008-02-04 2009-08-06 Kytos Biosystems S.A. Methods and compositions for use of crl 5803 cells for expression of biotherapeutics and encapsulated cell-based delivery
CA2716793A1 (en) 2008-03-05 2009-09-11 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of eg5 and vegf genes
FI20080326A0 (fi) 2008-04-30 2008-04-30 Licentia Oy Neurotroofinen tekijä MANF ja sen käytöt
US8052970B2 (en) * 2008-06-30 2011-11-08 The Regents Of The University Of Michigan Lysosomal phospholipase A2 (LPLA2) activity as a diagnostic and therapeutic target for identifying and treating systemic lupus erythematosis
EP3208337A1 (de) 2008-09-02 2017-08-23 Alnylam Pharmaceuticals, Inc. Zusammensetzungen zur kombinationshemmung der expression eines mutanten egfr und il-6
JP5529142B2 (ja) 2008-09-25 2014-06-25 アルナイラム ファーマシューティカルズ， インコーポレイテッド 血清アミロイドａ遺伝子の発現を阻害するための脂質製剤組成物および方法
US8168775B2 (en) 2008-10-20 2012-05-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin
NO2356135T3 (de) 2008-11-05 2018-03-24
SG171952A1 (en) 2008-12-04 2011-07-28 Opko Ophthalmics Llc Compositions and methods for selective inhibition of pro-angiogenic vegf isoforms
AU2009324534B2 (en) 2008-12-10 2015-07-30 Alnylam Pharmaceuticals, Inc. GNAQ targeted dsRNA compositions and methods for inhibiting expression
WO2010099341A1 (en) 2009-02-26 2010-09-02 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of mig-12 gene
US20100267806A1 (en) 2009-03-12 2010-10-21 David Bumcrot LIPID FORMULATED COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF Eg5 AND VEGF GENES
CA2755870C (en) 2009-03-20 2019-04-09 Angioblast Systems, Inc. Production of reprogrammed pluripotent cells
JP5766179B2 (ja) 2009-04-27 2015-08-19 ノバルティス アーゲー 筋肉増殖を増加させるための組成物および方法
EP2424987B1 (de) * 2009-05-01 2017-11-15 CuRNA, Inc. Behandlung von hämoglobin (hbf / hbg) verwandten krankheiten durch die inhibition des natürlichen antisense transcripts zu hbf / hbg
GB0908425D0 (en) 2009-05-15 2009-06-24 Medical Res Council Medical use
US9050276B2 (en) 2009-06-16 2015-06-09 The Trustees Of Columbia University In The City Of New York Autism-associated biomarkers and uses thereof
WO2011020023A2 (en) 2009-08-14 2011-02-17 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of a gene from the ebola virus
CA2778678A1 (en) 2009-10-30 2011-05-05 Cns Therapeutics, Inc. Improved neurturin molecules
WO2011053991A2 (en) 2009-11-02 2011-05-05 The Regents Of The University Of California Vault complexes for cytokine delivery
US8551781B2 (en) 2009-11-19 2013-10-08 The Regents Of The University Of California Vault complexes for facilitating biomolecule delivery
JP2013512677A (ja) * 2009-12-04 2013-04-18 オプコ オプサルミクス、エルエルシー Ｖｅｇｆの阻害のための組成物および方法
ES2893199T3 (es) 2010-03-29 2022-02-08 Alnylam Pharmaceuticals Inc Terapia de ARNbc para amiloidosis ocular relacionada con transtiretina (TTR)
JP5988961B2 (ja) 2010-04-28 2016-09-07 ザ ジェイ． デヴィッド グラッドストーン インスティテューツ 心筋細胞を発生させるための方法
SG185481A1 (en) 2010-05-10 2012-12-28 Univ California Endoribonuclease compositions and methods of use thereof
WO2011153323A2 (en) 2010-06-02 2011-12-08 Alnylam Pharmaceuticals, Inc. Compositions and methods directed to treating liver fibrosis
WO2011154553A2 (en) 2010-06-11 2011-12-15 Cellartis Ab Novel micrornas for the detection and isolaton of human embryonic stem cell-derived cardiac cell types
EA201390085A1 (ru) 2010-07-09 2013-06-28 Экселиксис, Инк. Композиции ингибиторов киназ для лечения рака
GB201012651D0 (en) 2010-07-28 2010-09-15 Univ Edinburgh Peptides
WO2012025873A2 (en) 2010-08-23 2012-03-01 Wyeth Llc STABLE FORMULATIONS OF NEISSERIA MENINGITIDIS rLP2086 ANTIGENS
PE20140173A1 (es) 2010-09-10 2014-02-20 Wyeth Llc Variantes no lipidadas de antigenos orf2086 de neisseria meningitidis
CN103391784A (zh) 2010-10-15 2013-11-13 纽约市哥伦比亚大学理事会 肥胖症-相关的基因和它们的蛋白和其用途
HUE045869T2 (hu) 2010-11-02 2020-01-28 Univ Columbia Módszerek hajhullásos rendellenességek kezelésére
ES2716819T3 (es) 2010-11-05 2019-06-17 Univ Leland Stanford Junior Opsinas quiméricas activadas por luz y métodos de uso de las mismas
CN103476456B (zh) 2010-11-05 2017-10-03 斯坦福大学托管董事会 奖赏相关行为的光遗传学控制
US9198975B2 (en) 2010-12-01 2015-12-01 The University Of North Carolina At Chapel Hill Methods and compositions for targeting sites of neovascular growth
EP2649182A4 (de) 2010-12-10 2015-05-06 Alnylam Pharmaceuticals Inc Zusammensetzungen und verfahren zur erhöhung einer erythropoietin (epo)-herstellung
WO2012079046A2 (en) 2010-12-10 2012-06-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of klf-1 and bcl11a genes
WO2012109495A1 (en) 2011-02-09 2012-08-16 Metabolic Solutions Development Company, Llc Cellular targets of thiazolidinediones
KR102481317B1 (ko) 2011-03-29 2022-12-26 알닐람 파마슈티칼스 인코포레이티드 Tmprss6 유전자의 발현을 억제하기 위한 조성물 및 방법
WO2012142526A1 (en) 2011-04-14 2012-10-18 Modiano Jaime Use of tumor fas expression to determine response to anti-cancer therapy
CA2840175C (en) 2011-05-19 2021-11-16 The Regents Of The University Of Michigan Integrin alpha-2 binding agents and use thereof to inhibit cancer cell proliferation
EP2714906A1 (de) 2011-06-01 2014-04-09 INSERM (Institut National de la Santé et de la Recherche Médicale) Verfahren zur einstellung der expression von mitochondrialem genom durch microrna
KR102540778B1 (ko) 2011-06-21 2023-06-07 알닐람 파마슈티칼스 인코포레이티드 아포리포단백질 c-iii(apoc3) 유전자의 발현 억제를 위한 조성물 및 방법
MX345095B (es) 2011-06-21 2017-01-17 Alnylam Pharmaceuticals Inc Composiciones de arni similares a angiopoyetina 3 (angptl3) y metodos para su uso.
CA2839896A1 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Assays and methods for determining activity of a therapeutic agent in a subject
US9068184B2 (en) 2011-06-21 2015-06-30 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of protein C (PROC) genes
EP3597750B1 (de) 2011-06-23 2022-05-04 Alnylam Pharmaceuticals, Inc. Serpina1-sirnas: materialzusammensetzungen und behandlungsverfahren
US20140328811A1 (en) 2011-08-01 2014-11-06 Alnylam Pharmaceuticals, Inc. Method for improving the success rate of hematopoietic stem cell transplants
AU2012308240A1 (en) 2011-09-15 2013-05-02 Los Angeles Biomedical Research Institute At Harbor-Ucla Medical Center Immunotherapy and diagnosis of mucormycosis using CotH
EP2766500A4 (de) 2011-10-14 2015-10-14 Univ Ohio State Verfahren und materialien in verbindung mit eierstockkrebs
US20140288156A1 (en) 2011-10-27 2014-09-25 Inserm (Institut National De La Sante Et De La Recherche Medicale) Methods for the treatment and diagnosis of atherosclerosis
CN104321069A (zh) 2011-10-27 2015-01-28 威尔斯达眼科制剂公司 编码视杆细胞来源的视锥细胞活力因子的载体
DK2785683T3 (da) 2011-11-30 2020-04-14 Ludwig Inst For Cancer Res Ltd Inkt-cellemodulatorer og fremgangsmåder til anvendelse heraf
GB201120860D0 (en) 2011-12-05 2012-01-18 Cambridge Entpr Ltd Cancer immunotherapy
US9365628B2 (en) 2011-12-16 2016-06-14 The Board Of Trustees Of The Leland Stanford Junior University Opsin polypeptides and methods of use thereof
RU2659423C2 (ru) 2012-02-16 2018-07-02 ЭйТИР ФАРМА, ИНК. ГИСТИДИЛ-тРНК-СИНТЕТАЗЫ ДЛЯ ЛЕЧЕНИЯ АУТОИММУННЫХ И ВОСПАЛИТЕЛЬНЫХ ЗАБОЛЕВАНИЙ
WO2013126521A1 (en) 2012-02-21 2013-08-29 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods for treating neurogenic disorders of the pelvic floor
SA115360586B1 (ar) 2012-03-09 2017-04-12 فايزر انك تركيبات لعلاج الالتهاب السحائي البكتيري وطرق لتحضيرها
SG10201602558UA (en) 2012-03-09 2016-05-30 Pfizer Neisseria meningitidis compositions and methods thereof
US10039777B2 (en) 2012-03-20 2018-08-07 Neuro-Lm Sas Methods and pharmaceutical compositions of the treatment of autistic syndrome disorders
PL2830662T3 (pl) 2012-03-29 2019-02-28 The Trustees Of Columbia University In The City Of New York Sposoby leczenia zaburzeń utraty włosów
US20150190532A1 (en) 2012-04-04 2015-07-09 The Trustees Of Columbia University In The City Of New York Smooth muscle specific inhibition for anti-restenotic therapy
WO2013153082A1 (en) 2012-04-10 2013-10-17 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the treatment of nonalcoholic steatohepatitis
US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
WO2013153139A1 (en) 2012-04-11 2013-10-17 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the treatment and diagnosis of acute leukemia
US9127274B2 (en) 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
EA038924B1 (ru) 2012-05-25 2021-11-10 Те Риджентс Оф Те Юниверсити Оф Калифорния Способы и композиции рнк-специфической модификации днк-мишени и рнк-специфической модуляции транскрипции
WO2013184209A1 (en) 2012-06-04 2013-12-12 Ludwig Institute For Cancer Research Ltd. Mif for use in methods of treating subjects with a neurodegenerative disorder
EP2869828B1 (de) 2012-07-09 2018-01-03 The Regents of The University of California Vault immuntherapie
WO2014014821A1 (en) 2012-07-19 2014-01-23 Redwood Biosciences, Inc. Antibody specific for cd22 and methods of use thereof
US20140037599A1 (en) * 2012-08-03 2014-02-06 The Trustees Of The University Of Pennsylvania Compositions and Methods of Treating T Cell Deficiency
MX359817B (es) 2012-08-16 2018-10-11 Ipierian Inc Anticuerpos que se enlazan a tau.
BR112015005091B1 (pt) 2012-09-06 2021-07-06 The University Of Chicago uso de um polinucleotídeo antissenso isolado
EP3722320A3 (de) 2012-10-25 2020-12-30 Bioverativ USA Inc. Anti-komplement-c1s-antikörper und verwendungen davon
JP6543572B2 (ja) 2012-11-02 2019-07-10 バイオベラティブ・ユーエスエイ・インコーポレイテッド 抗補体Ｃ１ｓ抗体とそれらの用途
EP2922476B1 (de) 2012-11-21 2019-10-09 Circuit Therapeutics, Inc. System für optogenetische therapie
KR102096014B1 (ko) 2012-12-05 2020-04-03 알닐람 파마슈티칼스 인코포레이티드 PCSK9 iRNA 조성물 및 그 사용 방법
EP2956557B1 (de) 2013-02-14 2018-07-11 The J. David Gladstone Institutes Zusammensetzungen und verfahren zur verwendung davon zur identifizierung von viruziden
PT3300745T (pt) 2013-02-15 2019-11-27 Univ California Recetor de antigénio quimérico e métodos de utilização do mesmo
US9745558B2 (en) 2013-02-18 2017-08-29 Vegenics Pty Limited VEGFR-3 ligand binding molecules and uses thereof
EP2964665B1 (de) 2013-03-08 2018-08-01 Pfizer Inc Immunogene fusionspolypeptide
EP2968613B1 (de) 2013-03-11 2019-09-11 University of Florida Research Foundation, Inc. Freisetzung von card-protein zur behandlung von augenentzündungen
WO2014160418A2 (en) 2013-03-13 2014-10-02 GeneWeave Biosciences, Inc. Non-replicative transduction particles and transduction particle-based reporter systems
US9481903B2 (en) 2013-03-13 2016-11-01 Roche Molecular Systems, Inc. Systems and methods for detection of cells using engineered transduction particles
KR102342916B1 (ko) 2013-03-14 2021-12-24 알닐람 파마슈티칼스 인코포레이티드 보체 성분 Ｃ5 ｉＲＮＡ 조성물 및 그 이용 방법
CA2907152A1 (en) 2013-03-15 2014-09-25 The Trustees Of Columbia University In The City Of New York Fusion proteins and methods thereof
EP2968997B1 (de) 2013-03-15 2019-06-26 The Board of Trustees of the Leland Stanford Junior University Optogenetische steuerung des verhaltenszustandes
US9937231B2 (en) 2013-03-27 2018-04-10 The General Hospital Corporation Methods and agents for treating Alzheimer's disease
EP2991491B1 (de) 2013-04-29 2019-12-25 The Board of Trustees of the Leland Stanford Junior University Vorrichtungen, systeme und verfahren zur optogenetischen modulation von wirkungspotentialen in zielzellen
EP3587578A1 (de) 2013-05-22 2020-01-01 Alnylam Pharmaceuticals, Inc. Tmprss6-irna-zusammensetzungen und verfahren zur verwendung davon
PT2999785T (pt) 2013-05-22 2018-07-09 Alnylam Pharmaceuticals Inc Composições de irna de serpina1 e métodos de uso das mesmas
WO2014191630A2 (en) 2013-05-28 2014-12-04 Helsingin Yliopisto Non-human animal model encoding a non-functional manf gene
CN111205369B (zh) 2013-05-29 2024-03-08 维拜昂公司 改变亨廷顿蛋白突变体降解的单链胞内抗体
US10166300B2 (en) 2013-06-04 2019-01-01 Virginia Commonwealth University Tripartite cancer theranostic nucleic acid constructs
EP3003022B1 (de) 2013-06-04 2017-11-22 Virginia Commonwealth University Verwendung eines trunkierten ccn1-promotors für krebsdiagnostik, therapeutika und theranostika
US9701985B2 (en) 2013-06-04 2017-07-11 Virginia Commonwealth University mda-9/syntenin promoter to image and treat metastatic cancer cells
WO2014197599A1 (en) 2013-06-04 2014-12-11 The Johns Hopkins University Peg-prom mediated surface expression of avidin/streptavidin
EP3004153B1 (de) 2013-06-04 2019-10-30 Virginia Commonwealth University Rekombinantes cytokin für eine krebstherapie
PL3007726T3 (pl) 2013-06-10 2021-01-11 Ipierian, Inc. Metody leczenia tauopatii
WO2014205511A1 (en) 2013-06-25 2014-12-31 University Of Canberra Methods and compositions for modulating cancer stem cells
CN105829538A (zh) 2013-08-14 2016-08-03 小利兰·斯坦福大学托管委员会 用于控制疼痛的组合物和方法
CA2923129C (en) 2013-09-08 2020-06-09 Pfizer Inc. Neisseria meningitidis compositions and methods thereof
US10124001B2 (en) 2013-09-18 2018-11-13 University Of Canberra Stem cell modulation II
WO2015050990A1 (en) 2013-10-02 2015-04-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
US10119143B2 (en) 2013-10-04 2018-11-06 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
WO2015071474A2 (en) 2013-11-18 2015-05-21 Crispr Therapeutics Ag Crispr-cas system materials and methods
EP3079727A1 (de) 2013-12-12 2016-10-19 INSERM - Institut National de la Santé et de la Recherche Médicale Verfahren zur vorbeugung und behandlung von diabetischer kardiomyopathie mithilfe von mir-424/322
CN105814205B (zh) 2013-12-12 2019-11-19 阿尔尼拉姆医药品有限公司 补体成分iRNA组合物及其使用方法
EP3080266B1 (de) 2013-12-12 2021-02-03 The Regents of The University of California Verfahren und zusammensetzungen zur modifizierung einer einzelsträngigen zielnukleinsäure
US9682123B2 (en) 2013-12-20 2017-06-20 The Trustees Of Columbia University In The City Of New York Methods of treating metabolic disease
EA201691587A1 (ru) 2014-02-11 2017-01-30 Элнилэм Фармасьютикалз, Инк. КОМПОЗИЦИИ НА ОСНОВЕ iRNA ДЛЯ КЕТОГЕКСОКИНАЗЫ (KHK) И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
US11053291B2 (en) 2014-02-19 2021-07-06 University Of Florida Research Foundation, Incorporated Delivery of Nrf2 as therapy for protection against reactive oxygen species
WO2015148974A2 (en) 2014-03-28 2015-10-01 The Board Of Trustees Of The Leland Stanford Junior University Engineered light-activated anion channel proteins and methods of use thereof
TW201607559A (zh) 2014-05-12 2016-03-01 阿尼拉製藥公司 治療ｓｅｒｐｉｎｃ１相關疾患之方法和組成物
KR20220087576A (ko) 2014-05-22 2022-06-24 알닐람 파마슈티칼스 인코포레이티드 안지오텐시노겐 (ａｇｔ) ｉｒｎａ 조성물 및 이의 사용 방법
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
EP3157552B1 (de) 2014-06-18 2019-10-23 Albert Einstein College of Medicine Syntac-polypeptide und verwendungen davon
US10800830B2 (en) 2014-08-08 2020-10-13 The Board Of Trustees Of The Leland Stanford Junior University High affinity PD-1 agents and methods of use
AU2015309686B2 (en) 2014-08-25 2020-05-14 Epiaxis Therapeutics Pty Ltd Compositions for modulating cancer stem cells and uses therefor
TW201614073A (en) 2014-08-25 2016-04-16 Geneweave Biosciences Inc Non-replicative transduction particles and transduction particle-based reporter systems
WO2016037163A1 (en) 2014-09-07 2016-03-10 Selecta Biosciences, Inc. Methods and compositions for attenuating gene therapy anti-viral transfer vector immune responses
DK3198018T3 (da) 2014-09-24 2021-03-01 Hope City Vektorvarianter af adenoassocieret virus til højeffektiv genomredigering og fremgangsmåder dertil
JOP20200115A1 (ar) 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))
JP6401871B2 (ja) 2014-11-05 2018-10-10 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． パーキンソン病の治療のためのａａｄｃポリヌクレオチド
JOP20200092A1 (ar) 2014-11-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها
EP3218484A4 (de) 2014-11-14 2018-05-30 Voyager Therapeutics, Inc. Zusammensetzungen und verfahren zur behandlung von amyotropher lateralsklerose (als)
SG11201703419UA (en) 2014-11-14 2017-05-30 Voyager Therapeutics Inc Modulatory polynucleotides
EP3221451A1 (de) 2014-11-17 2017-09-27 Alnylam Pharmaceuticals, Inc. Apolipoprotein-c3 (apoc3)-irna-zusammensetzungen und verfahren zur verwendung davon
EP3230441A4 (de) 2014-12-12 2018-10-03 Voyager Therapeutics, Inc. Zusammensetzungen und verfahren zur herstellung von scaav
EP3778644A3 (de) 2014-12-23 2021-06-02 The Trustees of Columbia University in the City of New York Fgfr-tacc fusionsproteine und verfahren dafür
JP2018510621A (ja) 2015-02-13 2018-04-19 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． パタチン様ホスホリパーゼドメイン含有３（ＰＮＰＬＡ３）ｉＲＮＡ組成物およびその使用方法
BR112017017460A2 (pt) 2015-02-19 2018-04-10 Pfizer Inc. composições de neisseria meningitidis e métodos das mesmas
SG11201707393SA (en) 2015-03-13 2017-10-30 Jackson Lab A three-component crispr/cas complex system and uses thereof
RS63956B1 (sr) 2015-04-06 2023-02-28 Bioverativ Usa Inc Humanizovana anti-c1s antitela i metode njihove upotrebe
US10669596B2 (en) 2015-04-07 2020-06-02 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Methods for inducing cell division of postmitotic cells
CA2981960C (en) 2015-04-08 2023-09-19 The University Of Chicago Compositions and methods for correcting limb girdle muscular dystrophy type 2c using exon skipping
WO2016191418A1 (en) 2015-05-26 2016-12-01 Salk Institute For Biological Studies Motor neuron-specific expression vectors
US10392607B2 (en) 2015-06-03 2019-08-27 The Regents Of The University Of California Cas9 variants and methods of use thereof
WO2016201301A1 (en) 2015-06-12 2016-12-15 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
WO2017023861A1 (en) 2015-08-03 2017-02-09 The Regents Of The University Of California Compositions and methods for modulating abhd2 activity
EP3798234A1 (de) 2015-09-02 2021-03-31 Immutep S.A.S. Agonistische anti-lag-3 antikörper
EP3347486A4 (de) 2015-09-09 2019-06-19 The Trustees of Columbia University in the City of New York Reduktion von er-mam-lokalisierten app-c99 und verfahren zur behandlung der alzheimer-krankheit
CA2998287A1 (en) 2015-09-24 2017-04-20 Crispr Therapeutics Ag Novel family of rna-programmable endonucleases and their uses in genome editing and other applications
US10188750B1 (en) 2015-10-23 2019-01-29 University Of South Florida Self-replicating cell selective gene delivery compositions, methods, and uses thereof
US10983110B2 (en) 2015-12-02 2021-04-20 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
SG11201804038VA (en) 2016-01-08 2018-06-28 Univ California Conditionally active heterodimeric polypeptides and methods of use thereof
CA3011894A1 (en) 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
CA3014774A1 (en) 2016-02-17 2017-08-24 Children's Medical Center Corporation Ffa1 (gpr40) as a therapeutic target for neural angiogenesis diseases or disorders
WO2017151940A2 (en) 2016-03-03 2017-09-08 Cue Biopharma, Inc. T-cell modulatory multimeric polypeptides and methods of use thereof
US10596274B2 (en) 2016-03-19 2020-03-24 Exuma Biotech Corp. Methods and compositions for transducing lymphocytes and regulated expansion thereof
US11325948B2 (en) 2016-03-19 2022-05-10 Exuma Biotech Corp. Methods and compositions for genetically modifying lymphocytes to express polypeptides comprising the intracellular domain of MPL
WO2020047527A2 (en) 2018-09-02 2020-03-05 F1 Bioventures, Llc Methods and compositions for genetically modifying lymphocytes in blood or in enriched pbmcs
US11111505B2 (en) 2016-03-19 2021-09-07 Exuma Biotech, Corp. Methods and compositions for transducing lymphocytes and regulating the activity thereof
WO2018161064A1 (en) 2017-03-03 2018-09-07 F1 Oncology, Inc. Methods and compositions for transducing and expanding lymphocytes and regulating the activity thereof
BR112018070357A2 (pt) 2016-04-04 2019-01-29 Bioverativ Usa Inc anticorpos anticomplemento contra o fator bb e seus usos
WO2017182981A1 (en) 2016-04-20 2017-10-26 Washington University Ppar agonist or lxr agonist for use in the treatment of systemic lupus erythematosus by modulation of lap activity
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3448874A4 (de) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. Zusammensetzungen zur behandlung einer erkrankung
EP3458588A4 (de) 2016-05-18 2020-01-15 Voyager Therapeutics, Inc. Modulatorische polynukleotide
WO2017201258A1 (en) 2016-05-18 2017-11-23 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
CA3019005A1 (en) 2016-05-18 2017-11-23 Cue Biopharma, Inc. T-cell modulatory multimeric polypeptides and methods of use thereof
JP2019522466A (ja) 2016-05-18 2019-08-15 アルバート アインシュタイン カレッジ オブ メディシン，インコーポレイティド 変異ｐｄ−ｌ１ポリペプチド、ｔ細胞調節多量体ポリペプチド、及びそれらの使用方法
EP3469083A1 (de) 2016-06-10 2019-04-17 Alnylam Pharmaceuticals, Inc. Gegen komplementkomponente c5 gerichtete irna-zusammensetzungen und verfahren zur verwendung davon zur behandlung von paroxysmaler nächtlicher hämoglobinurie (pnh)
EP3472318B1 (de) 2016-07-08 2023-09-06 Exuma Biotech Corp. Verfahren und zusammensetzungen zur transduktion von lymphozyten und regulierung der aktivität davon
EP3831281A1 (de) 2016-08-30 2021-06-09 The Regents of The University of California Verfahren für biomedizinisches targeting und freisetzung sowie vorrichtungen und systeme zur ausführung davon
US11780895B2 (en) 2016-09-13 2023-10-10 The Jackson Laboratory Targeted DNA demethylation and methylation
WO2018057648A1 (en) 2016-09-20 2018-03-29 The Board Of Trustees Of The Leland Stanford Junior University Peptide regulators of mitochondrial fusion and methods of use
EP3299460A1 (de) 2016-09-26 2018-03-28 Johann Wolfgang Goethe-Universität Frankfurt am Main Neuartige verbindungen und verfahren zur modulation der ubiquitinierung
MX2019004259A (es) 2016-10-12 2019-09-27 Bioverativ Usa Inc Anticuerpos anti-c1s y metodos de uso de los mismos.
US11154591B2 (en) 2016-10-14 2021-10-26 The Trustees Of Columbia University In The City Of New York Methods of treating alcohol abuse disorder
US11332713B2 (en) 2016-11-16 2022-05-17 KSQ Therapeutics, Inc. Gene-regulating compositions and methods for improved immunotherapy
SG10201913552UA (en) 2016-12-16 2020-03-30 Alnylam Pharmaceuticals Inc Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions
CA3043630A1 (en) 2016-12-22 2018-06-28 Cue Biopharma, Inc. T-cell modulatory multimeric polypeptides and methods of use thereof
EP3565829A4 (de) 2017-01-09 2021-01-27 Cue Biopharma, Inc. T-zell-modulierende multimere polypeptide und verfahren zur verwendung davon
BR112019014615A2 (pt) 2017-01-18 2020-06-02 F1 Oncology, Inc. Receptores de antígeno quimérico contra axl ou ror2 e métodos de uso dos mesmos
US20190367876A1 (en) 2017-01-18 2019-12-05 F1 Oncology, Inc. Methods of transducing and expanding immune cells and uses thereof
AU2018215585B2 (en) 2017-01-31 2022-03-17 Pfizer Inc. Neisseria meningitidis compositions and methods thereof
WO2018204803A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
JP2020518258A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
GB201710973D0 (en) 2017-07-07 2017-08-23 Avacta Life Sciences Ltd Scaffold proteins
US11497576B2 (en) 2017-07-17 2022-11-15 Voyager Therapeutics, Inc. Trajectory array guide system
EP3808849A1 (de) 2017-08-03 2021-04-21 Voyager Therapeutics, Inc. Zusammensetzungen und verfahren zur aav-freisetzung
KR20200064085A (ko) 2017-09-07 2020-06-05 큐 바이오파마, 인크. 접합 부위를 갖는 t-세포 조절 다량체 폴리펩타이드 및 이의 사용 방법
CN111315773A (zh) 2017-09-08 2020-06-19 马弗里克治疗公司 含有Fc区的条件性活化的结合部分
PE20212205A1 (es) 2017-09-08 2021-11-18 Maverick Therapeutics Inc Proteinas de union condicionalmente activadas restringidas
JP2021509253A (ja) 2017-10-10 2021-03-25 ナントバイオ，インコーポレイテッド ウイルス生成ペイロードに対して低い毒性を有する改変ｅｃ７細胞
TWI823868B (zh) 2017-10-11 2023-12-01 美商生物維瑞提夫美國公司 誘導補體活性之方法
AU2018352236A1 (en) 2017-10-16 2020-04-23 The Curators Of The University Of Missouri Treatment of amyotrophic lateral sclerosis (ALS)
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
KR20200095462A (ko) * 2017-10-18 2020-08-10 시티 오브 호프 Hbb 유전자 기능 회복을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법
WO2019089922A1 (en) 2017-11-01 2019-05-09 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
WO2019100039A1 (en) 2017-11-20 2019-05-23 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
CA3084825A1 (en) 2017-12-14 2019-06-20 Crispr Therapeutics Ag Novel rna-programmable endonuclease systems and their use in genome editing and other applications
US11008602B2 (en) 2017-12-20 2021-05-18 Roche Molecular Systems, Inc. Non-replicative transduction particles and transduction particle-based reporter systems
EP3737689A4 (de) 2018-01-09 2021-12-01 Cue Biopharma, Inc. Multimere t-zell-modulierende polypeptide und verfahren zur verwendung davon
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
US11667949B2 (en) 2018-02-15 2023-06-06 The Trustees Of Princeton University Reporter construct and biosensor for interferon second messenger 2-5A
EP3755795A4 (de) 2018-02-19 2022-07-20 Homology Medicines, Inc. Adeno-assoziierte viruszusammensetzungen zur wiederherstellung der f8-genfunktion und verfahren zur verwendung davon
BR112020018620A2 (pt) 2018-03-15 2020-12-29 KSQ Therapeutics, Inc. Composições de regulação gênica e métodos para imunoterapia aprimorada
AU2019239957A1 (en) 2018-03-19 2020-09-10 Bayer Healthcare Llc Novel RNA-programmable endonuclease systems and uses thereof
JP2021533744A (ja) 2018-08-09 2021-12-09 マベリック セラピューティクス, インコーポレイテッドMaverick Therapeutics, Inc. 条件的活性化型結合タンパク質を共発現及び精製するための方法
US20210348162A1 (en) 2018-08-16 2021-11-11 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
CN110857440B (zh) 2018-08-23 2021-02-19 武汉纽福斯生物科技有限公司 重组人ⅱ型线粒体动力蛋白样gtp酶基因序列及其应用
JP7450945B2 (ja) 2018-08-30 2024-03-18 テナヤ セラピューティクス， インコーポレイテッド ミオカルディンおよびａｓｃｌ１を用いた心細胞リプログラミング
CN111118016B (zh) 2018-10-30 2022-04-15 上海朗昇生物科技有限公司 治疗视网膜色素变性疾病的基因治疗载体
WO2020104649A2 (en) 2018-11-23 2020-05-28 Sanofi Novel rna compositions and methods for inhibiting angptl8
TW202039542A (zh) 2018-12-19 2020-11-01 美商庫爾生物製藥有限公司 多聚體ｔ細胞調節多肽及其使用方法
WO2020136154A1 (en) 2018-12-27 2020-07-02 F. Hoffmann-La Roche Ag Non-replicative transduction particles and transduction particle-based reporter systems for detection of acinetobacter baumannii
US11572595B2 (en) 2018-12-31 2023-02-07 Roche Molecular Systems, Inc. Non-replicative transduction particles with one or more non-native tail fibers and transduction particle-based reporter systems
US20220144949A1 (en) 2019-03-05 2022-05-12 Takeda Pharmaceutical Limited Company CONDITIONALLY ACTIVATED BINDING PROTEINS CONTAINING Fc REGIONS AND MOIETIES TARGETING TUMOR ANTIGENS
JP2022524338A (ja) 2019-03-05 2022-05-02 武田薬品工業株式会社 拘束され、条件的に活性化された結合タンパク質
EP3935080A4 (de) 2019-03-06 2023-04-05 Cue Biopharma, Inc. T-zell-modulierende multimere polypeptide und verfahren zur verwendung davon
MX2021010559A (es) 2019-03-07 2021-12-15 Univ California Polipéptidos efectores de crispr-cas y métodos de uso de estos.
US20220145274A1 (en) 2019-03-12 2022-05-12 Crispr Therapeutics Ag Novel high fidelity rna-programmable endonuclease systems and uses thereof
CN111718418B (zh) 2019-03-19 2021-08-27 华东师范大学 一种增强基因编辑的融合蛋白及其应用
JP2022527860A (ja) 2019-04-02 2022-06-06 ケンジョッケティ バイオテクノロジー，インク． 排出ポンプ－癌抗原マルチ特異性抗体ならびにそれに関する組成物、試薬、キットおよび方法
WO2020212586A1 (en) 2019-04-18 2020-10-22 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the treatment and prognosis of cancer
EP3736330A1 (de) 2019-05-08 2020-11-11 European Molecular Biology Laboratory Partikel mit modifiziertem adeno-assoziiertem virus (aav) zur gentherapie
CN112180094A (zh) 2019-07-04 2021-01-05 上海东慈生物科技有限公司 Dkk1抑制剂在预防和/或治疗肿瘤恶病质与糖尿病伴随疾病中的应用
US20230137971A1 (en) 2019-07-11 2023-05-04 Tenaya Therapeutics Inc. Cardiac cell reprogramming with micrornas and other factors
EP4003508A1 (de) 2019-07-31 2022-06-01 Memorial Sloan Kettering Cancer Center Perfusionsmodulierte tumordosismodellierung mit einzeldosis-strahlentherapie
EP4013788A1 (de) 2019-08-12 2022-06-22 Purinomia Biotech, Inc. Verfahren und zusammensetzungen zur förderung und verstärkung von t-zell-vermittelten immunantworten durch adcc-targeting von cd39-exprimierenden zellen
WO2021032069A1 (en) 2019-08-16 2021-02-25 Center For Excellence In Brain Science And Intelligence Technology, Chinese Academy Of Sciences Treatment of neuronal diseases
CN115176011A (zh) 2019-08-27 2022-10-11 赛诺菲 用于抑制pcsk9的组合物和方法
CN114616331A (zh) 2019-09-03 2022-06-10 阿尔尼拉姆医药品有限公司 抑制lect2基因表达的组合物和方法
WO2021067747A1 (en) 2019-10-04 2021-04-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing ugt1a1 gene expression
WO2021074695A1 (en) 2019-10-16 2021-04-22 Avacta Life Sciences Limited PD-L1 INHIBITOR - TGFβ INHIBITOR BISPECIFIC DRUG MOIETIES.
US20230040920A1 (en) 2019-11-01 2023-02-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
BR112022009216A2 (pt) 2019-11-13 2022-08-02 Alnylam Pharmaceuticals Inc Métodos e composições para tratar um distúrbio associado a angiotensinogênio (agt)
WO2021113736A1 (en) 2019-12-05 2021-06-10 Massachusetts Institute Of Technology Single-domain antibody to chloramphenicol
EP4085156B1 (de) 2019-12-31 2023-10-25 F. Hoffmann-La Roche AG Quantitatives pcr-screening von induzierbaren prophagen aus bakterienisolaten
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
WO2021154941A1 (en) 2020-01-31 2021-08-05 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions for use in the treatment of amyotrophic lateral sclerosis (als)
KR20220140593A (ko) 2020-02-10 2022-10-18 알닐람 파마슈티칼스 인코포레이티드 Vegf-a 발현을 사일런싱하기 위한 조성물 및 방법
AU2021230476A1 (en) 2020-03-02 2022-10-20 Tenaya Therapeutics, Inc. Gene vector control by cardiomyocyte-expressed microRNAs
GB202004498D0 (en) 2020-03-27 2020-05-13 Ucl Business Ltd Activity-dependent gene therapy for neurological disorders
US20230190785A1 (en) 2020-03-30 2023-06-22 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajc15 gene expression
EP4163375A4 (de) 2020-03-30 2024-01-03 Wuhan Neurophth Biotechnology Ltd Company Expressionsvektor des menschlichen nuklearfaktor-e2-verwandten faktors 2 und anwendung des expressionsvektors
US20230295622A1 (en) 2020-04-06 2023-09-21 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing myoc expression
JP2023521094A (ja) 2020-04-07 2023-05-23 アルナイラム ファーマシューティカルズ， インコーポレイテッド Ｓｃｎ９ａ発現をサイレンシングするための組成物および方法
EP4153746A1 (de) 2020-05-21 2023-03-29 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und verfahren zur hemmung der marc1-genexpression
JP2023528417A (ja) 2020-06-04 2023-07-04 ケンジョッケティ バイオテクノロジー，インク． Ａｂｃｇ２排出ポンプ－がん抗原多重特異性抗体並びにそれらに関連する組成物、試薬、キット、及び方法
GB202101299D0 (en) 2020-06-09 2021-03-17 Avacta Life Sciences Ltd Diagnostic polypetides and methods
GB202010981D0 (en) 2020-07-16 2020-09-02 Ucl Business Ltd Gene therapy for neuromuscular and neuromotor disorders
US20230295615A1 (en) 2020-08-07 2023-09-21 The Jackson Laboratory Targeted Sequence Insertion Compositions and Methods
US11781156B2 (en) 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
JP2023545502A (ja) 2020-10-16 2023-10-30 サノフイ リポタンパク質（ａ）を阻害するためのｒｎａ組成物および方法
JP2023546103A (ja) 2020-10-16 2023-11-01 サノフイ Ａｎｇｐｔｌ３を阻害するための新規のｒｎａ組成物および方法
AU2021365822A1 (en) 2020-10-21 2023-06-08 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
TW202233584A (zh) 2020-11-11 2022-09-01 歐洲分子生物學實驗室 用於基因治療之經修飾病毒顆粒
CA3201452A1 (en) 2020-12-01 2022-06-09 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
WO2022187289A1 (en) 2021-03-01 2022-09-09 Exuma Biotech Corp. Methods and compositions for the delivery of retroviral particles
KR20240004764A (ko) 2021-04-30 2024-01-11 칼리버 임뮤노쎄라퓨틱스, 인크. 변형된 mhc 발현을 위한 종양용해성 바이러스
WO2022234003A1 (en) 2021-05-07 2022-11-10 Avacta Life Sciences Limited Cd33 binding polypeptides with stefin a protein
EP4101928A1 (de) 2021-06-11 2022-12-14 Bayer AG Programmierbare typ-v-rna-endonukleasesysteme
IL308896A (en) 2021-06-11 2024-01-01 Bayer Ag Programmable type V RNA endoclase systems
KR20240026203A (ko) 2021-06-30 2024-02-27 알닐람 파마슈티칼스 인코포레이티드 안지오텐시노겐(agt)-관련 장애를 치료하는 방법 및 조성물
EP4144841A1 (de) 2021-09-07 2023-03-08 Bayer AG Neue programmierbare rna-endonuklease-systeme mit verbesserter pam-spezifität und deren verwendung
WO2023057567A1 (en) 2021-10-07 2023-04-13 Avacta Life Sciences Limited Pd-l1 binding affimers
WO2023057946A1 (en) 2021-10-07 2023-04-13 Avacta Life Sciences Limited Serum half-life extended pd-l1 binding polypeptides
WO2023102388A1 (en) 2021-11-30 2023-06-08 Sanofi Pasteur Inc. Human metapneumovirus viral vector-based vaccines
WO2023114658A1 (en) 2021-12-13 2023-06-22 Kenjockety Biotechnology, Inc. Anti-abcb1 antibodies
WO2023118068A1 (en) 2021-12-23 2023-06-29 Bayer Aktiengesellschaft Novel small type v rna programmable endonuclease systems
WO2023131682A1 (en) 2022-01-06 2023-07-13 Ucl Business Ltd Endogenous gene regulation to treat neurological disorders and diseases
GB202201744D0 (en) 2022-02-10 2022-03-30 Ucl Business Ltd Treatment of acquired focal epilepsy
WO2023159220A1 (en) 2022-02-18 2023-08-24 Kenjockety Biotechnology, Inc. Anti-cd47 antibodies
WO2023168305A1 (en) 2022-03-01 2023-09-07 Exuma Biotech Corp. Viral particles with membrane-bound hyaluronidase
WO2023222829A1 (en) 2022-05-17 2023-11-23 Centre Hospitalier Universitaire Vaudois Designed biosensors for enhanced t cell therapy
WO2023237587A1 (en) 2022-06-10 2023-12-14 Bayer Aktiengesellschaft Novel small type v rna programmable endonuclease systems
EP4299733A1 (de) 2022-06-30 2024-01-03 Inari Agriculture Technology, Inc. Zusammensetzungen, systeme und verfahren zum genomeditieren
WO2024005864A1 (en) 2022-06-30 2024-01-04 Inari Agriculture Technology, Inc. Compositions, systems, and methods for genome editing
EP4299739A1 (de) 2022-06-30 2024-01-03 Inari Agriculture Technology, Inc. Zusammensetzungen und verfahren zur genomeditierung
WO2024005863A1 (en) 2022-06-30 2024-01-04 Inari Agriculture Technology, Inc. Compositions, systems, and methods for genome editing
WO2024044659A1 (en) 2022-08-24 2024-02-29 Tectonic Therapeutic, Inc. Constitutively active g protein-coupled receptor compositions and methods of use thereof
WO2024068996A1 (en) 2022-09-30 2024-04-04 Centre Hospitalier Universitaire Vaudois (C.H.U.V.) Anti-sars-cov-2 antibodies and use thereof in the treatment of sars-cov-2 infection

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) * 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
WO1988008450A1 (en) * 1987-05-01 1988-11-03 Birdwell Finlayson Gene therapy for metabolite disorders
WO1991018088A1 (en) * 1990-05-23 1991-11-28 The United States Of America, Represented By The Secretary, United States Department Of Commerce Adeno-associated virus (aav)-based eucaryotic vectors
US5173414A (en) * 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors

Also Published As

Publication number Publication date
EP0566732B1 (de) 2003-04-02
KR930703453A (ko) 1993-11-30
CA2098483C (en) 2000-03-14
JP2003174896A (ja) 2003-06-24
ES2192555T3 (es) 2003-10-16
AU3134093A (en) 1993-06-07
ATE236263T1 (de) 2003-04-15
JPH06504680A (ja) 1994-06-02
IL103677A0 (en) 1993-04-04
PT101040A (pt) 1994-05-31
CA2098483A1 (en) 1993-05-09
DE69232983T2 (de) 2003-11-13
AU657829B2 (en) 1995-03-23
DK0566732T3 (da) 2003-07-21
US5252479A (en) 1993-10-12
EP0566732A1 (de) 1993-10-27
PT101040B (pt) 2003-06-30
WO1993009239A1 (en) 1993-05-13

Similar Documents

Publication Publication Date Title
DE69232983T2 (de) Adeno-assozierter virus 2 basalvektoren
AU2790292A (en) Viral recombinant vectors for expression in muscle cells
DE69128893D1 (de) Genetische veränderung von endothelzellen
HK1156069A1 (zh) 用作體外和體內 轉染與基因治療載體的完整微細胞
BR9914643A (pt) Vetores adeno-associados para expressão dofator viii por células alvo
IL173399A (en) Circular dna molecules, processes for their preparation, pharmaceutical compositions comprising them and use thereof in the manufacture of medical products for producing recombinant proteins in vivo
NL189092C (nl) Hybride menselijke leukocytinterferons, dubbelstrengs dna dat hiervoor codeert, expressievector, transformant micro-organisme, werkwijzen ter bereiding daarvan en farmaceutisch preparaat.
EP1007637A4 (de) Methoden zur erhöhung der effizienz rekombinanter aav produkte
IL80407A0 (en) Novel plant plasmid vector
Weatherall Scope and limitations of gene therapy
ATE284969T1 (de) Rekombinante adenovirale vektoren, die eine spleissequenz enthalten
ATE243741T1 (de) Zufuhr von genprodukten mittels mesangium-zellen
HUP0002913A2 (hu) Eljárások és készítmények szekretált fehérjéket, például béta-interferont kódoló géneket alkalmazó terápiára
DK0870044T3 (da) Reguleret proteinekspression i stabilt transfekterede pattedyrsceller
EP0235113A2 (de) Immortalisation von primären Zellen
AU657144B2 (en) Recombinant Marek's disease virus, process for preparing the same and vaccine containing the same
Eck The prospects for gene therapy
HUP9800520A2 (hu) Génexpresszió emlős sejtekben
Zhao Gene Transfer and Drug Delivery by Electronic Pulse Delivery: A Nonviral Delivery Approach
AU5276296A (en) Medicament containing at least one part of the ul84 protein of the cytomegalovirus, use of polypeptides corresponding to the amino acid sequence of the ul84 protein, and process for introducing ul84 into target cells
MXPA04007934A (es) Microorganismo envuelto.
AU2002359572A8 (en) 15603, a human ion channel family member

Legal Events

Date Code Title Description
8364 No opposition during term of opposition
8339 Ceased/non-payment of the annual fee