PT101040A - Vector seguro para a terapia com genes e metodo para a sua utilizacao - Google Patents

Vector seguro para a terapia com genes e metodo para a sua utilizacao

Info

Publication number
PT101040A
PT101040A PT101040A PT10104092A PT101040A PT 101040 A PT101040 A PT 101040A PT 101040 A PT101040 A PT 101040A PT 10104092 A PT10104092 A PT 10104092A PT 101040 A PT101040 A PT 101040A
Authority
PT
Portugal
Prior art keywords
gene therapy
genes
therapy
vectors
safe
Prior art date
Application number
PT101040A
Other languages
English (en)
Other versions
PT101040B (pt
Inventor
Arun Srivastava
Original Assignee
Res 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 Res Corp Technologies Inc filed Critical Res Corp Technologies Inc
Publication of PT101040A publication Critical patent/PT101040A/pt
Publication of PT101040B publication Critical patent/PT101040B/pt

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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Diabetes (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Virology (AREA)
  • Mycology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Emergency Medicine (AREA)
  • Endocrinology (AREA)
  • Obesity (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
PT101040A 1991-11-08 1992-11-06 Vector seguro para a terapia com genes PT101040B (pt)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US07/789,917 US5252479A (en) 1991-11-08 1991-11-08 Safe vector for gene therapy

Publications (2)

Publication Number Publication Date
PT101040A true PT101040A (pt) 1994-05-31
PT101040B PT101040B (pt) 2003-06-30

Family

ID=25149101

Family Applications (1)

Application Number Title Priority Date Filing Date
PT101040A PT101040B (pt) 1991-11-08 1992-11-06 Vector seguro para a terapia com genes

Country Status (13)

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

Families Citing this family (610)

* 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
CA2144848A1 (en) * 1992-09-18 1994-03-31 H. Michael Shepard 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
ES2310580T3 (es) 1993-04-30 2009-01-16 Wellstat Biologics Corporation Composiciones purificadas del virus de la enfermedad de newcastle.
US6686200B1 (en) * 1993-08-31 2004-02-03 Uab Research Foundation Methods and compositions for the large scale production of recombinant adeno-associated virus
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
US5834441A (en) * 1993-09-13 1998-11-10 Rhone-Poulenc Rorer Pharmaceuticals Inc. Adeno-associated viral (AAV) liposomes and methods related thereto
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
JPH10501686A (ja) 1994-04-13 1998-02-17 ザ ロックフェラー ユニヴァーシティ 神経系の細胞へのｄｎａのａａｖ仲介送達
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
US5658785A (en) * 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US20020159979A1 (en) 1994-06-06 2002-10-31 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
DE69521002T3 (de) 1994-08-12 2010-02-25 The University Of Utah Research Foundation, Salt Lake City Mutationen des mit 17q verbundenen Ovarial- und Brustkrebs Empfindlichkeitsgens
EP0699754B2 (en) 1994-08-12 2009-08-12 The University of Utah Research Foundation Method for diagnosing a predisposition for breast and ovarian cancer
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
CA2203809C (en) 1994-10-28 2008-06-03 James M. Wilson Recombinant adenovirus and methods of use thereof
US5856152A (en) * 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
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
US6638762B1 (en) 1994-11-28 2003-10-28 Genetic Therapy, Inc. Tissue-vectors specific replication and gene expression
US5998205A (en) 1994-11-28 1999-12-07 Genetic Therapy, Inc. Vectors for tissue-specific replication
FR2731710B1 (fr) * 1995-03-14 1997-04-30 Rhone Poulenc Rorer Sa Virus recombinants exprimant la lecithine cholesterol acyltransferase et utilisations en therapie genique
WO1996028560A1 (en) * 1995-03-16 1996-09-19 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
ES2187648T3 (es) * 1995-05-16 2003-06-16 Karl Tryggvason Metodo para la administracion de farmacos para terapia genica.
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
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
US6187757B1 (en) 1995-06-07 2001-02-13 Ariad Pharmaceuticals, Inc. Regulation of biological events using novel compounds
US20020068049A1 (en) * 1998-09-10 2002-06-06 Henderson Daniel R. Tissue specific adenoviral vectors
JPH11510046A (ja) * 1995-07-21 1999-09-07 ローヌ−プーラン ローラー ファーマシューティカルズ インコーポレイテッド アデノ関連ウイルスリポソーム及び樹状細胞をトランスフェクトして特異性免疫を刺激することにおけるそれらの使用
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
CN101028523A (zh) * 1995-11-30 2007-09-05 德克萨斯州立大学董事会 诊断和治疗肿瘤的方法及组合物
US5837492A (en) 1995-12-18 1998-11-17 Myriad Genetics, Inc. Chromosome 13-linked breast cancer susceptibility gene
CA2239733C (en) 1995-12-18 2001-04-03 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.
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
US5858351A (en) 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
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
AU3130597A (en) * 1996-05-16 1997-12-05 Duke University Tristetraprolin
US5780447A (en) * 1996-06-14 1998-07-14 St. Jude Children's Research Hospital Recombinant adeno-associated viral vectors
US6534314B1 (en) 1996-06-14 2003-03-18 Massachusetts Institute Of Technology Methods and compositions for transforming cells
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
AU737243B2 (en) 1996-07-03 2001-08-16 Merial, Inc. Recombinant canine adenovirus (CAV) containing exogenous DNA
US20020037867A1 (en) * 1999-02-26 2002-03-28 James M. Wilson Method for recombinant adeno-associated virus-directed gene therapy
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
EP0933997A4 (en) 1996-09-06 2000-08-16 Chiron Corp METHODS AND COMPOSITIONS FOR LIVER-SPECIFIC ADMINISTRATION OF THERAPEUTIC MOLECULES USING RECOMBINANT AAV VECTORS
AU4266597A (en) 1996-09-11 1998-04-14 General Hospital Corporation, The 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
CA2274146C (en) * 1996-12-05 2007-02-06 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
AU753781B2 (en) 1997-04-28 2002-10-31 Aventis Pharma S.A. Adenovirus-mediated intratumoral delivery of an angiogenesis antagonist for the treatment of tumors
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
WO1999018227A1 (en) * 1997-10-08 1999-04-15 Advanced Research And Technology Institute Chimeric parvovirus-based recombinant vector system that specifically targets the erythroid lineage
US6346415B1 (en) * 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
EP1027069B1 (en) 1997-10-29 2006-07-26 Genzyme Corporation Gene therapy for gaucher disease
BR9813930A (pt) 1997-11-06 2006-12-19 Chiron Spa antìgeno neisserial
CA2316808C (en) 1998-01-08 2007-04-10 Aventis Pharmaceuticals Products Inc. Transgenic rabbit that expresses a functional human lipoprotein (a)
US6780609B1 (en) 1998-10-23 2004-08-24 Genome Therapeutics Corporation High bone mass gene of 1.1q13.3
ATE446368T1 (de) 1998-01-14 2009-11-15 Novartis Vaccines & Diagnostic 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
MX343752B (es) 1998-05-01 2016-11-22 Novartis Vaccines & Diagnostics Inc Antigenos de neisseria meningitidis y composiciones que los contienen.
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
US6200560B1 (en) * 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated 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
AU6794700A (en) 1999-08-20 2001-03-19 Board Of Regents, The University Of Texas System Methods and compositions relating to hdac 4 and 5 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
PT2275551E (pt) 1999-10-29 2015-06-29 Novartis Vaccines & Diagnostic Péptidos antigénicos de neisseria
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
PT1232264E (pt) 1999-11-18 2009-11-26 Novartis Vaccines & Diagnostic Gene fgf-21 humano e produtos da expressão do gene
US6241710B1 (en) 1999-12-20 2001-06-05 Tricardia Llc Hypodermic needle with weeping tip and method of use
ES2588917T3 (es) 2000-01-17 2016-11-07 Glaxosmithkline Biologicals Sa Vacuna de VME suplementada contra meningococo
EP1854476A3 (en) 2000-02-09 2008-05-07 Bas Medical, Inc. Use of relaxin to treat diseases related to vasoconstriction
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 (en) 2000-05-31 2008-07-30 Novartis Vaccines and Diagnostics, Inc. Compositions and methods for treating neoplastic disease using chemotherapy and radiation sensitizers
US7198924B2 (en) 2000-12-11 2007-04-03 Invitrogen Corporation Methods and compositions for synthesis of nucleic acid molecules using multiple recognition sites
US7939087B2 (en) 2000-10-27 2011-05-10 Novartis Vaccines And Diagnostics, Inc. 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
EP1349924A2 (en) 2000-11-03 2003-10-08 Dana Farber Cancer Institute Methods and compositions for the diagnosis of cancer susceptibilites 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
BR0116756A (pt) 2000-12-28 2005-01-04 Wyeth Corp Proteìna protetora recombinante de streptococcus pneumoniae e uso da mesma
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
US8034791B2 (en) 2001-04-06 2011-10-11 The University Of Chicago Activation of Egr-1 promoter by DNA damaging chemotherapeutics
US20040242523A1 (en) * 2003-03-06 2004-12-02 Ana-Farber Cancer Institue And The Univiersity Of Chicago Chemo-inducible cancer gene therapy
WO2002081642A2 (en) 2001-04-06 2002-10-17 Georgetown University Gene brcc-3 and diagnostic and therapeutic uses thereof
ATE541937T1 (de) * 2001-04-06 2012-02-15 Univ Chicago Chemotherapeutische einleitung der egr-1-promoter-aktivität in gentherapie
AU2002303261A1 (en) 2001-04-06 2002-10-21 Georgetown University Gene brcc2 and diagnostic and therapeutic uses thereof
WO2002081641A2 (en) 2001-04-06 2002-10-17 Georgetown University Gene scc-112 and diagnostic and therapeutic uses thereof
US8067156B2 (en) 2001-05-31 2011-11-29 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
WO2003002746A2 (en) * 2001-06-29 2003-01-09 Novartis Ag Perv screening method and use thereof
MX339524B (es) 2001-10-11 2016-05-30 Wyeth Corp Composiciones inmunogenicas novedosas para la prevencion y tratamiento de enfermedad meningococica.
ES2386386T3 (es) 2001-12-12 2012-08-20 Novartis Vaccines And Diagnostics S.R.L. Inmunización contra Chlamydia trachomatis
CA2479118A1 (en) 2002-03-15 2003-09-25 Wyeth Holdings Corporation Mutants of the p4 protein of nontypable haemophilus influenzae with reduced enzymatic activity
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
US7138512B2 (en) 2002-04-10 2006-11-21 Georgetown University Gene SHINC-2 and diagnostic and therapeutic uses thereof
US20050222061A1 (en) * 2002-04-18 2005-10-06 Schulte Ralf W Means and methods for the specific inhibition of genes in cells and tissue of the cns and/or eye
ATE356871T1 (de) * 2002-05-17 2007-04-15 Alligator Bioscience Ab Eine methode zur in vitro molekularen evolution der proteinfunktion
MXPA04011624A (es) 2002-05-24 2005-03-07 Schering Corp Anticuerpo neutralizante humano anti-igfr.
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
ATE546528T1 (de) 2002-11-13 2012-03-15 Univ Jefferson Zusammensetzungen und verfahren zur diagnose und behandlung von krebs
JP4754219B2 (ja) 2002-12-02 2011-08-24 アムジエン・フレモント・インコーポレイテツド 腫瘍壊死因子を対象とする抗体、およびそれらの使用
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
JP2007524349A (ja) 2003-01-16 2007-08-30 ザ・トラスティーズ・オブ・ザ・ユニバーシティ・オブ・ペンシルバニア ＩＣＡＭ−１のｓｉＲＮＡ阻害のための組成物及び方法
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
BR0318307A (pt) * 2003-05-21 2006-07-11 Ares Trading Sa polipeptìdeo, molécula de ácido nucleico purificado, vetor, célula hospedeira, ligante, composto, método para diagnosticar uma doença em um paciente, uso de um polipeptìdeo, composição farmacêutica, composição de vacina, métodos para tratar uma doença para monitorar o tratamento terapêutico de doença em um paciente, método para a identificação de um composto que é efetivo no tratamento e/ou diagnóstico de doença, kit, e, animal não-humano transgênico ou knockout
CA2538843A1 (en) * 2003-09-12 2005-03-24 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
DE602004027538D1 (de) 2003-12-01 2010-07-15 Life Technologies Corp 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
EP1713938A2 (en) 2004-02-09 2006-10-25 Thomas Jefferson University DIAGNOSIS AND TREATMENT OF CANCERS WITH MicroRNA LOCATED IN OR NEAR CANCER-ASSOCIATED CHROMOSOMAL FEATURES
US7422741B2 (en) 2004-03-05 2008-09-09 Vegenics Limited VEGFR-3 fusion proteins
ES2423060T3 (es) * 2004-03-12 2013-09-17 Alnylam Pharmaceuticals, Inc. Agentes iRNA que tienen como diana al 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
KR101222281B1 (ko) 2004-03-29 2013-01-28 가르파마 컴퍼니 리미티드 신규 갈렉틴 9 개변체 단백질 및 그 용도
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
EP1781284B1 (en) * 2004-08-25 2010-10-13 The University of Chicago Use of the combination comprising temozolomide and tnf-alpha for treating glioblastoma
FI20050753A7 (fi) 2004-09-03 2006-03-04 Licentia Oy Uudet peptidit
GB0423126D0 (en) * 2004-10-18 2004-11-17 Ares Trading Sa Protein
WO2006055635A2 (en) * 2004-11-15 2006-05-26 Mount Sinai School Of Medicine Of New York University 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
EP3782655A1 (en) 2005-05-17 2021-02-24 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
WO2007008252A1 (en) * 2005-07-12 2007-01-18 Temple University - Of The Commonwealth Systems Of Higher Education 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
DK2594591T3 (en) 2005-08-11 2018-07-30 Matossian Rogers Arpi TCR-V-BETA RELATED PEPTIDES FOR TREATMENT AND DIAGNOSIS OF AUTOIMMUNE DISEASE
ES2363758T3 (es) 2005-08-15 2011-08-16 Vegenics Pty Ltd Vegf y pdgf modificados con propiedades angiogénicas mejoradas.
US8481505B2 (en) 2005-09-12 2013-07-09 The Ohio State University Research Foundation Compositions and methods for the diagnosis and therapy of BCL2-associated cancers
US7972813B2 (en) * 2005-09-30 2011-07-05 Vertex Pharmaceuticals Incorporated Tetrodotoxin-resistant sodium channel alpha subunit
US20080200408A1 (en) * 2005-09-30 2008-08-21 Mccormack Kenneth Deletion mutants of 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
PL1969003T3 (pl) 2005-12-14 2011-05-31 Herantis Pharma Plc Zastosowanie białka czynnika neurotroficznego
AU2006337093B2 (en) * 2005-12-22 2013-03-14 Opko Pharmaceuticals, Llc. Compositions and methods for regulating complement system
NZ569883A (en) 2005-12-28 2011-12-22 Translational Therapeutics Inc Use of triazole containing compounds for inhibiting 4E activity and proliferation of a cell
AU2007205234B2 (en) 2006-01-05 2012-07-12 The Ohio State University Research Foundation MicroRNA-based methods and compositions for the diagnosis, prognosis and treatment of lung cancer
EP2484782B1 (en) 2006-01-05 2015-11-18 The Ohio State University Research Foundation MicroRNA-based methods and compositions for the diagnosis and treatment of solid cancers
WO2007081680A2 (en) 2006-01-05 2007-07-19 The Ohio State University Research Foundation Microrna expression abnormalities in pancreatic endocrine and acinar tumors
US8229398B2 (en) * 2006-01-30 2012-07-24 Qualcomm Incorporated GSM authentication in a CDMA network
US20090214496A1 (en) * 2006-01-30 2009-08-27 Licentia Ltd. Bmx/etk tyrosine kinase gene therapy materials and methods
EP2522748A1 (en) 2006-03-02 2012-11-14 The Ohio State University MicroRNA expression profile associated with pancreatic cancer
CA2647769C (en) 2006-03-20 2015-05-26 Japan Science And Technology Agency Control of intracellular target molecule by ip3 receptor-binding protein
ES2446362T3 (es) 2006-03-20 2014-03-07 The Ohio State University Research Foundation Huellas de microARN durante megacariocipoyesis humana
JP5704741B2 (ja) 2006-03-31 2015-04-22 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． Ｅｇ５遺伝子発現の抑制のための組成物および方法
EP2007880A2 (en) 2006-04-07 2008-12-31 Neuro Therapeutics AB Survival and development of neural cells
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
US7605251B2 (en) 2006-05-11 2009-10-20 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the PCSK9 gene
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
EP2392583A1 (en) 2006-05-19 2011-12-07 Alnylam Europe AG. RNAi modulation of Aha and therapeutic uses thereof
US7888498B2 (en) * 2006-05-22 2011-02-15 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
DK2054431T3 (da) 2006-06-09 2012-01-02 Novartis Ag Konformere af bakterielle adhæsiner
JP2009540011A (ja) 2006-06-12 2009-11-19 エクセジェニックス、インク．ディー／ビー／エー オプコ ヘルス、インク． 血管新生のｓｉＲＮＡ阻害のための組成物及び方法
ES2425387T3 (es) 2006-07-13 2013-10-15 The Ohio State University Research Foundation Mir-106a para diagnosticar adenocarcinoma de colon de pronóstico de supervivencia pobre
MY151438A (en) 2006-07-28 2014-05-30 Sanofi Aventis Compositions and method for treatment of tumors
US7872118B2 (en) * 2006-09-08 2011-01-18 Opko Ophthalmics, Llc siRNA and methods of manufacture
US20090209478A1 (en) 2006-09-21 2009-08-20 Tomoko Nakayama Compositions and methods for inhibiting expression of the hamp gene
PL2068922T3 (pl) 2006-10-19 2012-11-30 Csl Ltd Przeciwciała anty-IL-13R alfa1 i ich zastosowania
ES2501947T3 (es) 2006-10-19 2014-10-02 Csl Limited Antagonistas de anticuerpos de alta afinidad del receptor alfa 1 de interleucina
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
CN106890342B (zh) * 2007-02-06 2022-09-23 俞泰俊 使用基因疗法治疗和预防神经变性疾病
US8841436B2 (en) * 2007-03-15 2014-09-23 University Hospitals Cleveland Medical Center Screening, diagnosing, treating and prognosis of pathophysiologic status 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
JP5350360B2 (ja) 2007-03-29 2013-11-27 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 エボラ由来の遺伝子の発現を阻害するための組成物および方法
EP2319926B1 (en) 2007-07-05 2016-08-31 Arrowhead Research Corporation 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
EP2173911B1 (en) 2007-07-31 2014-06-18 The Ohio State University Research Foundation Methods for reverting methylation by targeting dnmt3a and dnmt3b
EP2653561B1 (en) 2007-08-03 2016-03-02 The Ohio State University Research Foundation Ultraconserved regions encoding ncRNAs
EP3028708A1 (en) 2007-08-22 2016-06-08 The Ohio State University Research Foundation Methods and compositions for inducing deregulation of epha7 and erk phosphorylation in human acute leukemias
EP3138926A3 (en) 2007-09-06 2017-04-05 The Ohio State University Research Foundation Microrna signatures in human ovarian cancer
EP2212440A4 (en) 2007-10-11 2011-04-06 Univ Ohio State Res Found METHOD AND COMPOSITIONS FOR DIAGNOSIS AND TREATMENT OF ADENOCARCINOMES OF DISHES
FI20070808A0 (fi) 2007-10-25 2007-10-25 Mart Saarma GDNF:n silmukointivariantit ja niiden käytöt
WO2009076400A2 (en) 2007-12-10 2009-06-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of factor vii gene
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
CN105267233B (zh) 2008-03-05 2019-07-26 阿尔尼拉姆医药品有限公司 用于抑制Eg5和VEGF基因表达的组合物和方法
FI20080326A0 (fi) 2008-04-30 2008-04-30 Licentia Oy Neurotroofinen tekijä MANF ja sen käytöt
WO2010002895A2 (en) 2008-06-30 2010-01-07 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
EP2690175B1 (en) 2008-09-02 2016-12-28 Alnylam Pharmaceuticals, Inc. Compositions and methods for combined inhibition of mutant EGFR gene and IL-6 expression
US8546554B2 (en) 2008-09-25 2013-10-01 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of Serum Amyloid A gene
EP2937418B1 (en) 2008-10-20 2017-10-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin
AU2009313615B2 (en) 2008-11-05 2012-11-29 Regents Of The University Of Minnesota Multicomponent immunogenic composition for the prevention of beta-hemolytic streptococcal (BHS) disease
JP5832293B2 (ja) 2008-12-04 2015-12-16 オプコ ファーマシューティカルズ、エルエルシー 血管新生促進ｖｅｇｆイソ型を選択的に抑制する組成物および方法
EP2373382B1 (en) 2008-12-10 2017-02-15 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
CA2754043A1 (en) 2009-03-12 2010-09-16 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of eg5 and vegf genes
WO2010105311A1 (en) 2009-03-20 2010-09-23 Angioblast Systems, Inc. Production of reprogrammed pluripotent cells
ES2655877T3 (es) 2009-04-27 2018-02-22 Novartis Ag Composiciones y métodos para aumentar el crecimiento muscular
WO2010127195A2 (en) * 2009-05-01 2010-11-04 Curna, Inc. Antisense oligonucleotides of hemoglobins
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
US9029338B2 (en) 2009-08-14 2015-05-12 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of a gene from the ebola virus
BR112012009801A8 (pt) 2009-10-30 2016-08-30 Cns Therapeutics Inc Polipeptídeo neurturina e variante de neurturina
EP2496607B1 (en) 2009-11-02 2017-09-27 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
US8227444B2 (en) * 2009-12-04 2012-07-24 Opko Ophthalmics, Llc Compositions and methods for inhibition of VEGF
US20130281510A1 (en) 2010-03-29 2013-10-24 Kumamoto University siRNA Therapy for Transthyretin (TTR) Related Ocular Amyloidosis
EP3578657B1 (en) 2010-04-06 2024-03-20 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of cd274/pd-l1 gene
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
CA2798703A1 (en) 2010-05-10 2011-11-17 The Regents Of The University Of California Endoribonuclease compositions and methods of use thereof
EP3456827A3 (en) 2010-06-02 2019-05-08 Alnylam Pharmaceuticals, Inc. Compositions and methods directed to treating liver fibrosis
US20130150256A1 (en) 2010-06-11 2013-06-13 Jane Synnergren Novel micrornas for the detection and isolation of human embryonic stem cell-derived cardiac cell types
JP2013539453A (ja) 2010-07-09 2013-10-24 エクセリクシス， インク． 癌の治療のためのキナーゼ阻害物質の組み合わせ
GB201012651D0 (en) 2010-07-28 2010-09-15 Univ Edinburgh Peptides
CA2808975C (en) 2010-08-23 2018-10-30 Wyeth Llc Stable formulations of neisseria meningitidis rlp2086 antigens
KR101584871B1 (ko) 2010-09-10 2016-01-22 와이어쓰 엘엘씨 네이세리아 메닝기티디스 orf2086 항원의 비-지질화된 변이체
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
PT2635299T (pt) 2010-11-02 2019-10-14 Univ Columbia Métodos de tratamento de distúrbios de perda de cabelo
ES2625179T3 (es) 2010-11-05 2017-07-18 The Board Of Trustees Of The Leland Stanford Junior University Control optogenético de comportamientos relacionados con la recompensa
EP3486253A1 (en) 2010-11-05 2019-05-22 The Board of Trustees of The Leland Stanford Junior University Light-activated chimeric opsins and methods of using the same
EP2698635A3 (en) 2010-12-01 2014-05-28 The University Of North Carolina At Chapel Hill Methods and compositions for targeting sites of neovascular growth
US9193973B2 (en) 2010-12-10 2015-11-24 Alynylam Pharmaceuticals, Inc. Compositions and methods for increasing erythropoietin (EPO) production
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
KR102104401B1 (ko) 2011-03-29 2020-04-27 알닐람 파마슈티칼스 인코포레이티드 Tmprss6 유전자의 발현을 억제하기 위한 조성물 및 방법
US10352936B2 (en) 2011-04-14 2019-07-16 Apoplogic Pharmaceuticals, Inc. Use of tumor Fas expression to determine response to anti-cancer therapy
ES2748577T3 (es) 2011-05-19 2020-03-17 Univ Michigan Regents Agentes de fijación de la integrina alfa-2 y uso de los mismos para inhibir la proliferación de las células de cáncer
EP2714906A1 (en) 2011-06-01 2014-04-09 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for adjusting expression of mitochondrial genome by microrna
CA2839573A1 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof
EP2723351B1 (en) 2011-06-21 2018-02-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of protein c (proc) genes
EP2723756B1 (en) 2011-06-21 2020-03-11 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of apolipoprotein c-iii (apoc3) genes
CA2839896A1 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Assays and methods for determining activity of a therapeutic agent in a subject
US20140235693A1 (en) 2011-06-23 2014-08-21 Alnylam Pharmaceuticals, Inc. Serpina1 sirnas: compositions of matter and methods of treatment
JP2014526887A (ja) 2011-08-01 2014-10-09 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 造血幹細胞移植の成功率を改善する方法
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
CA2852066A1 (en) 2011-10-14 2013-04-18 The Ohio State University Methods and materials related to ovarian cancer
EP2771482A1 (en) 2011-10-27 2014-09-03 Institut National de la Sante et de la Recherche Medicale (INSERM) Methods for the treatment and diagnosis of atherosclerosis
CN110777124A (zh) 2011-10-27 2020-02-11 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
EP2785683B1 (en) 2011-11-30 2020-02-05 Ludwig Institute for Cancer Research Ltd. Inkt cell modulators and methods of using the same
GB201120860D0 (en) 2011-12-05 2012-01-18 Cambridge Entpr Ltd Cancer immunotherapy
CA2859364C (en) 2011-12-16 2019-05-07 The Board Of Trustees Of The Leland Stanford Junior University Opsin polypeptides and methods of use thereof
EP2814514B1 (en) 2012-02-16 2017-09-13 Atyr Pharma, Inc. Histidyl-trna synthetases for treating autoimmune and inflammatory diseases
ES2628006T3 (es) 2012-02-21 2017-08-01 Circuit Therapeutics, Inc. Composiciones para el tratamiento de trastornos neurogénicos del suelo pélvico
CA2865745C (en) 2012-03-09 2018-01-09 Pfizer Inc. Neisseria meningitidis compositions and methods thereof
SA115360586B1 (ar) 2012-03-09 2017-04-12 فايزر انك تركيبات لعلاج الالتهاب السحائي البكتيري وطرق لتحضيرها
US10039777B2 (en) 2012-03-20 2018-08-07 Neuro-Lm Sas Methods and pharmaceutical compositions of the treatment of autistic syndrome disorders
DK2830662T3 (da) 2012-03-29 2019-01-02 Univ Columbia Fremgangsmåder til behandlng af hårtabsforstyrrelser
WO2013152230A1 (en) 2012-04-04 2013-10-10 The Trustees Of Columbia University In The City Of New York Smooth muscle specific inhibition for anti-restenotic therapy
US9193971B2 (en) 2012-04-10 2015-11-24 Institut National De La Sante Et De La Recherche Medicale (Inserm) 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
ES2728782T3 (es) 2012-05-25 2019-10-28 Univ California Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN
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
US20150150821A1 (en) 2012-07-09 2015-06-04 The Regents Of The University Of California Vault Immunotherapy
CN104582717B (zh) 2012-07-19 2018-03-13 雷德伍德生物科技股份有限公司 对cd22有特异性的抗体及其使用方法
US20140037599A1 (en) * 2012-08-03 2014-02-06 The Trustees Of The University Of Pennsylvania Compositions and Methods of Treating T Cell Deficiency
EP2885010B1 (en) 2012-08-16 2020-01-01 Ipierian, Inc. Methods of treating a tauopathy
WO2014039916A1 (en) 2012-09-06 2014-03-13 The University Of Chicago Antisense polynucleotides to induce exon skipping and methods of treating dystrophies
WO2014066744A2 (en) 2012-10-25 2014-05-01 True North Therapeutics, Inc. Anti-complement c1s antibodies and uses thereof
ES2912396T3 (es) 2012-11-02 2022-05-25 Bioverativ Usa Inc Anticuerpos anti-C1s del complemento y uso de los mismos
CA2894718A1 (en) 2012-11-21 2014-05-30 Circuit Therapeutics, Inc. System and method for optogenetic therapy
BR112015013105B1 (pt) 2012-12-05 2022-02-08 Alnylam Pharmaceuticals, Inc Agente de rnai de fita dupla capaz de inibir a expressão de pcsk9, seus usos, composição farmacêutica e método de inibição da expressão de pcsk9 em uma célula in vitro
US10106817B2 (en) 2013-02-14 2018-10-23 The J. David Gladstone Institutes Compositions and methods of use thereof for identifying anti-viral agents
IL305629A (en) 2013-02-15 2023-11-01 Univ California Chimeric antigen receptor and methods of using it
MY170528A (en) 2013-02-18 2019-08-09 Vegenics Pty Ltd Vegfr-3 ligand binding molecules and uses thereof
CA2903716C (en) 2013-03-08 2019-04-09 Pfizer Inc. Immunogenic fusion polypeptides
US10981961B2 (en) 2013-03-11 2021-04-20 University Of Florida Research Foundation, Incorporated Delivery of card protein as therapy for occular inflammation
BR112015023108A8 (pt) 2013-03-13 2018-01-23 Geneweave Biosciences Inc sistema de acondicionamento de célula bacteriana, método para acondicionamento de uma molécula de ácido nucléico repórter, composição, método para detectar uma presença ou uma ausência de uma célula bacteriana em uma amostra, vetor, método para detectar um transcrito alvo em uma célula, método para detectar a presença de uma célula bacteriana em uma amostra e kit.
US9481903B2 (en) 2013-03-13 2016-11-01 Roche Molecular Systems, Inc. Systems and methods for detection of cells using engineered transduction particles
PT2970974T (pt) 2013-03-14 2017-11-29 Alnylam Pharmaceuticals Inc Composições de irna de componente do complemento c5 e métodos para a sua utilização
WO2014151734A1 (en) 2013-03-15 2014-09-25 The Trustees Of Columbia University In The City Of New York Fusion proteins and methods thereof
ES2742492T3 (es) 2013-03-15 2020-02-14 Univ Leland Stanford Junior Control optogenético del estado conductual
EP2978446B1 (en) 2013-03-27 2020-03-04 The General Hospital Corporation Anti-cd33 antibody for use in treating alzheimer's disease
US10220092B2 (en) 2013-04-29 2019-03-05 The Board Of Trustees Of The Leland Stanford Junior University Devices, systems and methods for optogenetic modulation of action potentials in target cells
AU2014268529C1 (en) 2013-05-22 2020-10-01 Alnylam Pharmaceuticals, Inc. TMPRSS6 iRNA compositions and methods of use thereof
TWI664187B (zh) 2013-05-22 2019-07-01 阿尼拉製藥公司 絲胺酸蛋白酶抑制因子Ａ１ ｉＲＮＡ組成物及其使用方法
WO2014191630A2 (en) 2013-05-28 2014-12-04 Helsingin Yliopisto Non-human animal model encoding a non-functional manf gene
EP3071598B1 (en) 2013-05-29 2021-07-07 Vybion, Inc. Single chain intrabodies that alter huntingtin mutant degradation
US9701985B2 (en) 2013-06-04 2017-07-11 Virginia Commonwealth University mda-9/syntenin promoter to image and treat metastatic cancer cells
US9951114B2 (en) 2013-06-04 2018-04-24 Virginia Commonwealth University Recombinant cancer therapeutic cytokine
WO2014209553A1 (en) 2013-06-04 2014-12-31 Virginia Commonwealth University Use of a truncated ccn1 promoter for cancer diagnostics, therapeutics and theranostics
US10166300B2 (en) 2013-06-04 2019-01-01 Virginia Commonwealth University Tripartite cancer theranostic nucleic acid constructs
WO2014197599A1 (en) 2013-06-04 2014-12-11 The Johns Hopkins University Peg-prom mediated surface expression of avidin/streptavidin
SMT202000401T1 (it) 2013-06-10 2020-11-10 Ipierian Inc Metodi di trattamento di una taupatia
SG11201510369WA (en) 2013-06-25 2016-01-28 Univ Canberra Methods and compositions for modulating cancer stem cells
JP6621747B2 (ja) 2013-08-14 2019-12-18 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 疼痛を制御するための組成物及び方法
RU2662968C2 (ru) 2013-09-08 2018-07-31 Пфайзер Инк. Иммуногенная композиция против neisseria meningitidis (варианты)
CA2923765C (en) 2013-09-18 2021-06-29 University Of Canberra Stem cell modulation ii
MX385871B (es) 2013-10-02 2025-03-18 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion del gen lect2.
WO2015051318A1 (en) 2013-10-04 2015-04-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the alas1 gene
EP3760719A1 (en) 2013-11-18 2021-01-06 CRISPR Therapeutics AG Crispr-cas system materials and methods
WO2015089368A2 (en) 2013-12-12 2015-06-18 Alnylam Pharmaceuticals, Inc. Complement component irna compositions and methods of use thereof
EP3079727A1 (en) 2013-12-12 2016-10-19 INSERM - Institut National de la Santé et de la Recherche Médicale Methods for the prevention and treatment of diabetic cardiomyopathy using mir-424/322
EP3835419A1 (en) 2013-12-12 2021-06-16 The Regents of The University of California Methods and compositions for modifying a single stranded target nucleic acid
US9682123B2 (en) 2013-12-20 2017-06-20 The Trustees Of Columbia University In The City Of New York Methods of treating metabolic disease
EP3105331B1 (en) 2014-02-11 2021-06-23 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
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
EP3581580A1 (en) 2014-03-28 2019-12-18 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
SG11201609376SA (en) 2014-05-22 2016-12-29 Alnylam Pharmaceuticals Inc Angiotensinogen (agt) irna compositions and methods of use thereof
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
JP6652932B2 (ja) 2014-06-18 2020-02-26 アルバート アインシュタイン カレッジ オブ メディシン ｓｙｎＴａｃポリペプチド及びその使用
EP3177640B1 (en) 2014-08-08 2020-05-06 The Board of Trustees of the Leland Stanford Junior University High affinity pd-1 agents and methods of use
KR101946180B1 (ko) 2014-08-25 2019-02-08 지니위브 바이오사이언시스, 인코포레이티드 비-복제 형질도입 입자 및 형질도입 입자-기반 리포터 시스템
AU2015309686B2 (en) 2014-08-25 2020-05-14 Epiaxis Therapeutics Pty Ltd Compositions for modulating cancer stem cells and uses therefor
EA201790551A1 (ru) 2014-09-07 2017-07-31 Селекта Байосайенсиз, Инк. Способы и композиции для ослабления иммунных ответов к пропускающему экзон вирусному трансферному вектору
WO2016049230A1 (en) 2014-09-24 2016-03-31 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
TWI864340B (zh) 2014-10-10 2024-12-01 美商艾爾妮蘭製藥公司 用於抑制hao1(羥酸氧化酶1(乙醇酸鹽氧化酶))基因表現的組合物及方法
EP3215191B1 (en) 2014-11-05 2024-08-14 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
TWI769565B (zh) 2014-11-10 2022-07-01 美商阿尼拉製藥公司 Ｂ型肝炎病毒（ＨＢＶ）ｉＲＮＡ組成物及其用途方法
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
CN114717264A (zh) 2014-11-14 2022-07-08 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
WO2016081444A1 (en) 2014-11-17 2016-05-26 Alnylam Pharmaceuticals, Inc. Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
EP3925979A3 (en) 2014-12-23 2022-03-23 The Trustees of Columbia University in the City of New York Fgfr-tacc fusion proteins and methods thereof
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
KR20190049940A (ko) 2015-02-19 2019-05-09 화이자 인코포레이티드 나이세리아 메닌지티디스 조성물 및 그의 방법
CA3091771C (en) 2015-03-13 2026-04-28 The Jackson Laboratory A three-component crispr/cas complex system and uses thereof
RS60762B1 (sr) * 2015-04-03 2020-10-30 Univ Massachusetts Oligonukleotidna jedinjenja za ciljane irnk hantingtina
EP3929293A3 (en) 2015-04-03 2022-03-16 University Of Massachusetts Fully stabilized asymmetric sirna
EP4212175A1 (en) 2015-04-06 2023-07-19 Bioverativ USA Inc. Humanized anti-c1s antibodies and methods of use thereof
WO2016164371A1 (en) 2015-04-07 2016-10-13 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Methods for inducing cell division of postmitotic cells
US10273483B2 (en) 2015-04-08 2019-04-30 The University Of Chicago Compositions and methods for correcting limb girdle muscular dystrophy type 2C using exon skipping
US10729790B2 (en) 2015-05-26 2020-08-04 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
EP3307316A1 (en) 2015-06-12 2018-04-18 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
EP3331536A4 (en) 2015-08-03 2019-03-27 The Regents of The University of California COMPOSITIONS AND METHODS OF MODULATING THE ABHD2 ACTIVITY
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
RU2760582C2 (ru) 2015-09-02 2021-11-29 Иммутеп С.А.С. Анти-LAG-3 антитела
EP3347486A4 (en) 2015-09-09 2019-06-19 The Trustees of Columbia University in the City of New York REDUCTION OF ER-MAM-LOCALIZED APP-C99 AND METHOD FOR THE TREATMENT OF ALZHEIMER DISEASE
WO2017064546A1 (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
AU2016362477A1 (en) 2015-12-02 2018-06-14 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
IL308717A (en) 2015-12-07 2024-01-01 Genzyme Corp Methods and compositions for treating a serpinc1-associated disorder
MX2018008416A (es) 2016-01-08 2019-11-11 Univ California Polipéptidos heterodímericos condicionalmente activos y métodos para su uso.
WO2017132669A1 (en) 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
AU2017221441A1 (en) 2016-02-17 2018-10-04 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
US12128102B2 (en) 2016-03-08 2024-10-29 Takeda Pharmaceutical Company Limited Constrained conditionally activated binding proteins
WO2020047527A2 (en) 2018-09-02 2020-03-05 F1 Bioventures, Llc Methods and compositions for genetically modifying lymphocytes in blood or in enriched pbmcs
WO2018161064A1 (en) 2017-03-03 2018-09-07 F1 Oncology, Inc. Methods and compositions for transducing and expanding lymphocytes and regulating the activity thereof
US11111505B2 (en) 2016-03-19 2021-09-07 Exuma Biotech, Corp. Methods and compositions for transducing lymphocytes and regulating the activity thereof
CN108884460B (zh) 2016-03-19 2023-04-28 埃克苏马生物技术公司 淋巴细胞转导及其扩增调节的方法与组合物
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
US12590321B2 (en) 2016-03-19 2026-03-31 Exuma Biotech Corp. Methods and compositions for genetically modifying and expanding lymphocytes and regulating the activity thereof
AU2017246794B2 (en) 2016-04-04 2024-07-04 Genzyme Corporation Anti-complement factor Bb antibodies and uses thereof
US20190145961A1 (en) 2016-04-20 2019-05-16 Washington University Ppar agonist or lxr agonist for use in the treatment of systemic lupus erythematosus by modulation of lap activity
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
SG11201808552XA (en) 2016-05-18 2018-10-30 Cue Biopharma Inc T-cell modulatory multimeric polypeptides and methods of use thereof
IL297576B2 (en) 2016-05-18 2024-02-01 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
US10584337B2 (en) 2016-05-18 2020-03-10 Voyager Therapeutics, Inc. Modulatory polynucleotides
IL262606B2 (en) 2016-05-18 2023-04-01 Albert Einstein College Medicine Inc pd-l1 variant polypeptides, T-cell modulatory multimeric polypeptides, and methods of using them
EP3469083A1 (en) 2016-06-10 2019-04-17 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
CA3030003A1 (en) 2016-07-08 2018-01-11 F1 Oncology, Inc. Methods and compositions for transducing lymphocytes and regulating the activity thereof
EP3831281B1 (en) 2016-08-30 2026-02-25 The Regents of The University of California Devices and systems for biomedical targeting and delivery
AU2017327384B2 (en) 2016-09-13 2021-05-20 The Jackson Laboratory Targeted enhanced DNA demethylation
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 (en) 2016-09-26 2018-03-28 Johann Wolfgang Goethe-Universität Frankfurt am Main Novel compounds and methods for modulating ubiquitination
IL326479A (en) 2016-10-12 2026-04-01 Bioverativ Usa Inc Anti-c1s antibodies and methods of use thereof
WO2018071814A1 (en) 2016-10-14 2018-04-19 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
TWI865997B (zh) 2016-12-16 2024-12-11 美商阿尼拉製藥公司 使用甲狀腺素運載蛋白（ＴＴＲ）ｉＲＮＡ組成物於治療或預防ＴＴＲ相關疾病之方法
CN110325205B (zh) 2016-12-22 2023-08-15 库尔生物制药有限公司 T细胞调节性多聚体多肽及其使用方法
US11851471B2 (en) 2017-01-09 2023-12-26 Cue Biopharma, Inc. T-cell modulatory multimeric polypeptides and methods of use thereof
EP3571294A1 (en) 2017-01-18 2019-11-27 F1 Oncology, Inc. Methods of transducing and expanding immune cells and uses thereof
SG10202107808SA (en) 2017-01-18 2021-08-30 F1 Oncology Inc Chimeric antigen receptors against axl or ror2 and methods of use thereof
SG11201906519RA (en) 2017-01-31 2019-08-27 Pfizer Neisseria meningitidis compositions and methods thereof
EP3579858A4 (en) 2017-02-07 2020-12-23 The Regents of The University of California GENE THERAPY AGAINST HAPLOINSUFFICIENCY
WO2018148454A1 (en) 2017-02-09 2018-08-16 The Regents Of The University Of California Chimeric t cell antigen receptors and methods of use thereof
CN110678190A (zh) 2017-03-28 2020-01-10 宾夕法尼亚大学董事会 保护移植的组织免受排斥的方法
MX2019013172A (es) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CN111065407A (zh) 2017-06-16 2020-04-24 普罗特利卡公司 纤连蛋白结合结构域嵌合抗原受体及其使用方法
WO2018237245A1 (en) 2017-06-23 2018-12-27 University Of Massachusetts TWO TAIL SELF-ADMINISTRATION SIRNA AND ASSOCIATED METHODS
GB201710973D0 (en) 2017-07-07 2017-08-23 Avacta Life Sciences Ltd Scaffold proteins
JP2020530442A (ja) 2017-07-10 2020-10-22 ジェンザイム・コーポレーション 血友病を有する対象の出血事象を処置するための方法および組成物
AU2018301829B2 (en) 2017-07-13 2024-08-01 Alnylam Pharmaceuticals, Inc. Methods for inhibition of HAO1 (hydroxyacid oxidase 1 (glycolate oxidase) gene expression
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
US11512327B2 (en) 2017-08-03 2022-11-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
WO2019051127A1 (en) 2017-09-07 2019-03-14 Cue Biopharma, Inc. MULTIMER MODULATOR POLYPEPTIDE OF LYMPHOCYTE T HAVING CONJUGATION SITES AND METHODS OF USE THEREOF
CN111356700A (zh) 2017-09-08 2020-06-30 马弗里克治疗公司 受约束的条件性活化的结合蛋白
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
MX2020003042A (es) 2017-09-29 2020-11-18 Voyager Therapeutics Inc Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.
CA3077594C (en) 2017-10-10 2022-11-29 Nantbio, Inc. Modified ec7 cells having low toxicity to viral production payloads
CN111527105B (zh) 2017-10-11 2024-10-25 美国比奥维拉迪维股份有限公司 诱导补体活性的方法
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
US12201657B2 (en) * 2017-10-18 2025-01-21 City Of Hope Adeno-associated virus compositions for restoring HBB gene function and methods of use thereof
WO2019089755A1 (en) 2017-10-31 2019-05-09 Western Oncolytics Ltd. Platform oncolytic vector for systemic delivery
CA3078971A1 (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
WO2019139896A1 (en) 2018-01-09 2019-07-18 Cue Biopharma, Inc. Multimeric t-cell modulatory polypeptides and methods of use thereof
JP2021511047A (ja) 2018-01-19 2021-05-06 ジェネレーション バイオ カンパニー 無細胞合成から得ることができる閉端ＤＮＡベクターおよびｃｅＤＮＡベクターを得るためのプロセス
AU2019215152A1 (en) 2018-02-01 2020-08-20 Homology Medicines, Inc. Adeno-associated virus compositions for restoring PAH gene function and methods of use thereof
WO2019152841A1 (en) 2018-02-01 2019-08-08 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
US11306329B2 (en) 2018-02-19 2022-04-19 City Of Hope Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof
CN112040986A (zh) 2018-03-15 2020-12-04 Ksq治疗公司 用于改进的免疫疗法的基因调控组合物和方法
JP7550648B2 (ja) 2018-03-19 2024-09-13 クリスパー セラピューティクス アーゲー 新規ｒｎａプログラム可能エンドヌクレアーゼ系およびその使用
US11447769B2 (en) 2018-03-27 2022-09-20 The Trustees Of The University Of Pennsylvania Modified immune cells having enhanced function and methods for screening for same
EP3773633A4 (en) 2018-04-06 2022-01-26 The Regents of The University of California METHODS OF TREATMENT OF GLIOBLASTOMA
CA3096202A1 (en) 2018-04-06 2019-10-10 The Regents Of The University Of California Methods of treating egfrviii expressing glioblastomas
WO2019200013A1 (en) 2018-04-11 2019-10-17 Cancer Targeting Systems, Inc. Therapeutic constructs for treating cancer
CN112424359A (zh) 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
JP2021524233A (ja) 2018-05-17 2021-09-13 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ 薬物耐性免疫細胞及びその用途
US12258395B2 (en) 2018-06-11 2025-03-25 University Of Florida Research Foundation, Incorporated Materials and methods for treating stress-related disorders and cancer
WO2020018166A1 (en) 2018-07-16 2020-01-23 The Regents Of The University Of Michigan Nuclease-mediated nucleic acid modification
EP3833444A1 (en) 2018-08-09 2021-06-16 Maverick Therapeutics, Inc. Coexpression and purification method of conditionally activated binding proteins
BR112021002440A2 (pt) 2018-08-10 2021-05-04 University Of Massachusetts oligonucleotídeos modificados visando snps
AR115960A1 (es) 2018-08-16 2021-03-17 Alnylam Pharmaceuticals Inc Composiciones y métodos para inhibir la expresión del gen lect2
EP3841205A4 (en) 2018-08-22 2022-08-17 The Regents of The University of California VARIANT TYPE V CRISPR/CAS EFFECTOR POLYPEPTIDES AND METHODS OF USE THEREOF
WO2020041769A1 (en) 2018-08-23 2020-02-27 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
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
KR20210102870A (ko) 2018-08-30 2021-08-20 테나야 테라퓨틱스, 인코포레이티드 미오카르딘 및 ascl1을 사용한 심장 세포 재프로그래밍
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
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
WO2020097445A1 (en) 2018-11-09 2020-05-14 Inari Agriculture, Inc. Rna-guided nucleases and dna binding proteins
CN113166760A (zh) 2018-11-23 2021-07-23 赛诺菲 用于抑制angptl8的新型rna组合物和方法
CA3121191A1 (en) 2018-11-28 2020-06-04 Crispr Therapeutics Ag Optimized mrna encoding cas9 for use in lnps
TWI856047B (zh) 2018-12-19 2024-09-21 美商信號生物製藥公司 多聚體ｔ細胞調節多肽及其使用方法
CN113227404A (zh) 2018-12-27 2021-08-06 豪夫迈·罗氏有限公司 用于检测鲍氏不动杆菌的非复制型转导颗粒和基于转导颗粒的报告系统
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
CN113614232A (zh) 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
JP2022524337A (ja) 2019-03-05 2022-05-02 武田薬品工業株式会社 腫瘍抗原を標的とするｆｃ領域及び部分を含有する条件的に活性化された結合タンパク質
EP4592313A3 (en) 2019-03-05 2025-11-19 Takeda Pharmaceutical Company Limited Constrained conditionally activated binding proteins
JP7691927B2 (ja) 2019-03-06 2025-06-12 キュー バイオファーマ， インコーポレイテッド Ｔ細胞調節抗原提示ポリペプチド及びその使用方法
WO2020181062A1 (en) 2019-03-06 2020-09-10 Cue Biopharma, Inc. T-cell modulatory multimeric polypeptides and methods of use thereof
EP4219700A1 (en) 2019-03-07 2023-08-02 The Regents of the University of California Crispr-cas effector polypeptides and methods of use thereof
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
CN114450411A (zh) 2019-04-01 2022-05-06 特纳亚治疗股份有限公司 具有工程化衣壳的腺相关病毒
WO2020206033A1 (en) 2019-04-02 2020-10-08 Kenjockety Biotechnology, Inc. Efflux pump-cancer antigen multi-specific antibodies and compositions, reagents, kits and methods related thereto
JP2022529917A (ja) 2019-04-18 2022-06-27 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル ガンの処置及び予後のための方法
EP3736330A1 (en) 2019-05-08 2020-11-11 European Molecular Biology Laboratory Modified adeno-associated virus (aav) particles for gene therapy
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
EP3997226A1 (en) 2019-07-11 2022-05-18 Tenaya Therapeutics, Inc. Cardiac cell reprogramming with micrornas and other factors
WO2021020979A1 (en) 2019-07-31 2021-02-04 Memorial Sloan Kettering Cancer Center Perfusion modulated tumor dose sculpting with single dose radiotherapy
KR20220047989A (ko) 2019-08-09 2022-04-19 유니버시티 오브 매사추세츠 Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드
CN114127121B (zh) 2019-08-12 2025-04-11 北京恩瑞尼生物科技股份有限公司 用于通过cd39表达细胞的adcc靶向促进和增强t细胞介导的免疫反应的方法和组合物
US12612657B2 (en) 2019-08-16 2026-04-28 The Jackson Laboratory Extrachromosomal DNA labeling
JP2022543722A (ja) 2019-08-16 2022-10-14 センター フォー エクセレンス イン ブレイン サイエンス アンド インテリジェンス テクノロジー，チャイニーズ アカデミー オブ サイエンシーズ 機能的ニューロン死に関連する神経系疾患の予防および／または治療におけるＰｔｂｐ１阻害剤の使用
CN115176011B (zh) 2019-08-27 2025-11-11 赛诺菲 用于抑制pcsk9的组合物和方法
US20220290152A1 (en) 2019-09-03 2022-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
WO2021062063A1 (en) 2019-09-26 2021-04-01 Chan Zuckerberg Biohub, Inc. Improved variants of tev protease for biotechnological applications
CA3155669A1 (en) 2019-09-27 2021-04-01 Pfizer Inc. Neisseria meningitidis compositions and methods thereof
US12503699B2 (en) 2019-10-04 2025-12-23 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.
FI4034170T3 (fi) 2019-10-23 2026-04-16 Cue Biopharma Inc Tgf-beta-polypeptidejä
US20230040920A1 (en) 2019-11-01 2023-02-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
IL292865A (en) 2019-11-13 2022-07-01 Alnylam Pharmaceuticals Inc Methods and preparations for the treatment of angiotensinogen-related disorder - (agt)
WO2021113736A1 (en) 2019-12-05 2021-06-10 Massachusetts Institute Of Technology Single-domain antibody to chloramphenicol
WO2021136752A1 (en) 2019-12-31 2021-07-08 F. Hoffmann-La Roche Ag Quantitative pcr screening of inducible prophage from bacterial isolates
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)
PH12022552080A1 (en) 2020-02-10 2024-02-12 Alnylam Pharmaceuticals Inc Compositions and methods for silencing vegf-a expression
CN115552022A (zh) 2020-03-02 2022-12-30 特纳亚治疗股份有限公司 由心肌细胞表达的微rna控制的基因载体
US12655229B2 (en) 2020-03-18 2026-06-16 Memorial Sloan Kettering Cancer Center Anti-ceramide antibodies
GB202004498D0 (en) 2020-03-27 2020-05-13 Ucl Business Ltd Activity-dependent gene therapy for neurological disorders
WO2021195876A1 (zh) 2020-03-30 2021-10-07 武汉纽福斯生物科技有限公司 一种人核因子e2相关因子2的表达载体及其应用
US20230190785A1 (en) 2020-03-30 2023-06-22 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajc15 gene expression
IL297121A (en) 2020-04-06 2022-12-01 Alnylam Pharmaceuticals Inc Compositions and methods for silencing myoc expression
AU2021251754A1 (en) 2020-04-07 2022-11-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing SCN9A expression
US12686725B2 (en) 2020-05-05 2026-07-21 Mythic Therapeutics, Inc. Anti-DLL3 antibodies and methods of use
WO2021237097A1 (en) 2020-05-21 2021-11-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
CA3174079A1 (en) 2020-05-26 2021-12-02 Anastasia Khvorova Synthetic oligonucleotides having regions of block and cluster modifications
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
CN116529267A (zh) 2020-06-04 2023-08-01 肯乔克蒂生物技术股份有限公司 Abcg2外排泵-癌症抗原多特异性抗体及其相关组合物、试剂、试剂盒和方法
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
JP7415042B2 (ja) 2020-10-09 2024-01-16 テナヤ セラピューティクス， インコーポレイテッド プラコフィリン２遺伝子治療の方法および組成物
EP4229201A1 (en) 2020-10-16 2023-08-23 Sanofi Rna compositions and methods for inhibiting lipoprotein(a)
US20230383294A1 (en) 2020-10-16 2023-11-30 Sanofi Novel rna compositions and methods for inhibiting angptl3
CA3198823A1 (en) 2020-10-21 2022-04-28 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
MX2023005446A (es) 2020-11-11 2023-07-18 European Molecular Biology Laboratory Particulas virales modificadas para terapia genica.
JP2023552289A (ja) 2020-11-19 2023-12-15 カリヴィル イムノセラピューティクス， インコーポレイテッド 腫瘍微小環境リモデリングによる腫瘍溶解性免疫療法
WO2022119873A1 (en) 2020-12-01 2022-06-09 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
AU2022229358A1 (en) 2021-03-01 2023-09-07 Exuma Biotech Corp. Methods and compositions for the delivery of retroviral particles
EP4329786A4 (en) 2021-04-30 2024-12-11 KaliVir Immunotherapeutics, Inc. Oncolytic viruses for modified mhc expression
WO2022234003A1 (en) 2021-05-07 2022-11-10 Avacta Life Sciences Limited Cd33 binding polypeptides with stefin a protein
MX2023014008A (es) 2021-05-27 2023-12-11 Alnylam Pharmaceuticals Inc Composiciones y metodos para silenciar la expresion de anhidrasa carbonica 2.
MX2023014774A (es) 2021-06-11 2024-01-15 Bayer Ag Nuevos sistemas de endonucleasas programables de arn tipo v.
EP4101928A1 (en) 2021-06-11 2022-12-14 Bayer AG Type v rna programmable endonuclease systems
IL309296A (en) 2021-06-30 2024-02-01 Alnylam Pharmaceuticals Inc Methods and compositions for the treatment of angiotensinogen-related disorder (AGT-)
TW202325311A (zh) 2021-08-31 2023-07-01 美商艾拉倫製藥公司 誘導細胞死亡之DFFA樣效應子B (CIDEB) iRNA組合物及其使用方法
EP4144841A1 (en) 2021-09-07 2023-03-08 Bayer AG Novel small rna programmable endonuclease systems with impoved pam specificity and uses thereof
TW202334196A (zh) 2021-10-07 2023-09-01 英商阿法克塔生命科學有限公司 Pd-l1結合多肽
WO2023057946A1 (en) 2021-10-07 2023-04-13 Avacta Life Sciences Limited Serum half-life extended pd-l1 binding polypeptides
EP4433512A4 (en) 2021-11-19 2025-12-31 Univ Pennsylvania Genetically modified pan-leukocyte-specific CD45 antigen to facilitate T-lymphocyte therapy
JP2024544054A (ja) 2021-11-30 2024-11-27 サノフィ パスツール インコーポレイテッド ヒトメタニューモウイルスのウイルスベクターに基づくワクチン
KR20240116829A (ko) 2021-12-13 2024-07-30 윌리엄 로버트 아라툰 리빙 트러스트 데이티드 어거스트 29, 2016 항-abcb1 항체
EP4453191A1 (en) 2021-12-23 2024-10-30 Bayer Aktiengesellschaft Novel small type v rna programmable endonuclease systems
US20250064974A1 (en) 2022-01-06 2025-02-27 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
US20250099612A1 (en) 2022-03-01 2025-03-27 Exuma Biotech Corp. Viral particles with membrane-bound hyaluronidase
CA3248580A1 (en) 2022-04-29 2023-11-02 Purinomia Biotech, Inc. METHODS AND COMPOSITIONS FOR THE TREATMENT OF DISEASES AND DISORDERS CAUSED BY EOSINOPHIL ACTIVITY
US20250262274A1 (en) 2022-05-17 2025-08-21 École Polytechnique Fédérale de Lausanne 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
WO2024005863A1 (en) 2022-06-30 2024-01-04 Inari Agriculture Technology, Inc. Compositions, systems, and methods for genome editing
WO2024005864A1 (en) 2022-06-30 2024-01-04 Inari Agriculture Technology, Inc. Compositions, systems, and methods for genome editing
EP4299739A1 (en) 2022-06-30 2024-01-03 Inari Agriculture Technology, Inc. Compositions, systems, and methods for genome editing
EP4299733A1 (en) 2022-06-30 2024-01-03 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
CN117947040A (zh) 2022-10-31 2024-04-30 苏州荷光科汇生物科技有限公司 用于目的基因的表达盒及其应用
US20260166072A1 (en) 2022-11-04 2026-06-18 Genkardia Inc. Oligonucleotide-based therapeutics targeting cyclin d2 for the treatment of heart failure
US20260109972A1 (en) 2022-11-09 2026-04-23 The Regents Of The University Of California High-Throughput Discovery of Multi-Partite CRISPR-Based Editors
WO2024105633A1 (en) 2022-11-18 2024-05-23 Kyoto Prefectural Public University Corporation Compositions for mitophagy induction and uses thereof
EP4619524A1 (en) 2022-11-18 2025-09-24 Genkardia Inc. Methods and compositions for preventing, treating, or reversing cardiac diastolic dysfunction
US20260209732A1 (en) 2022-12-13 2026-07-23 Bayer Aktiengesellschaft Engineered type v rna programmable endonucleases and their uses
KR20250141243A (ko) 2023-02-06 2025-09-26 블루록 테라퓨틱스 엘피 데그론 융합 단백질 및 그의 생산 및 사용 방법
WO2024178397A2 (en) 2023-02-24 2024-08-29 Elevatebio Technologies, Inc. Modified immune effector cells and methods of use
CN118576695A (zh) 2023-03-01 2024-09-03 上海三弹生物医药科技有限公司 一种治疗或改善恶病质的方法
CN120957741A (zh) 2023-03-13 2025-11-14 宾夕法尼亚大学董事会 用于肿瘤治疗的car修饰剂的产生
WO2024189583A1 (en) 2023-03-15 2024-09-19 Kyoto Prefectural Public University Corporation Peptide expression constructs and uses thereof
CN121285568A (zh) 2023-03-30 2026-01-06 制药五有限责任公司 编码视杆细胞源性视锥细胞活力因子和人IgK信号序列的载体
JP2026517843A (ja) 2023-05-03 2026-06-02 アイオーエックス セラピューティクス リミテッド Ｉｎｋｔ細胞調節剤リポソーム組成物及び使用方法
GB202307563D0 (en) 2023-05-19 2023-07-05 Univ King Abdullah Sci & Tech Methods and compositions
AU2024277678A1 (en) 2023-05-25 2025-11-27 Dispatch Biotherapeutics, Inc. Synthetic cancer antigens as targets for treating cancers
CN121712794A (zh) 2023-06-29 2026-03-20 迪斯派奇生物治疗公司 合成细胞因子受体
CN116790615B (zh) 2023-07-11 2024-11-22 康霖生物科技（杭州）有限公司 一种用于过敏性疾病的基因治疗载体核酸构建体及其使用方法
EP4748395A1 (en) 2023-07-19 2026-05-27 Shanghai Anyea Therapeutics Co., Ltd Therapy based on intracerebral phosphate homeostasis promoter
TW202519537A (zh) 2023-07-21 2025-05-16 義大利商博雷亞醫療有限責任公司 經化學遺傳表面修飾之病毒顆粒
AU2024324215A1 (en) 2023-08-11 2026-02-26 Abalytics Oncology, Inc. Anti-ctla-4 antibodies and related binding molecules and methods and uses thereof
CN118001292B (zh) 2023-09-01 2024-11-29 香港中文大学（深圳） 靶向rna的组合物及其用途
TW202521691A (zh) 2023-10-06 2025-06-01 美商藍岩醫療公司 經工程化之v型rna可程式核酸內切酶及其用途
WO2025090858A1 (en) 2023-10-27 2025-05-01 Biogen Ma Inc. Methods for identifying aav capsid variants with desired characteristics
WO2025111402A1 (en) 2023-11-21 2025-05-30 Board Of Regents Of The University Of Nebraska Anti-amyloid beta antibodies and related compositions and methods thereof
TW202540200A (zh) 2023-12-01 2025-10-16 美商基利科學股份有限公司 抗ｆａｐ－ｌｉｇｈｔ融合蛋白及其用途
AU2024406222A1 (en) 2023-12-20 2026-06-04 Bristol-Myers Squibb Company Antibodies targeting il-18 receptor beta (il-18rβ) and related methods
WO2025171383A2 (en) 2024-02-09 2025-08-14 Dispatch Biotherapeutics, Inc. Engineered cancer antigens and related methods and uses
WO2025171388A1 (en) 2024-02-09 2025-08-14 Dispatch Biotherapeutics, Inc. Engineered cancer antigens with modified domains and related methods and uses
TW202548024A (zh) 2024-03-19 2025-12-16 義大利商博雷亞醫療有限責任公司 靶向ｃｎｓ中之組織
WO2025199352A2 (en) 2024-03-20 2025-09-25 Juno Therapeutics, Inc. Antibodies specific for solute carrier family 34 member 2 (slc34a2)
WO2025240670A2 (en) 2024-05-15 2025-11-20 Abalytics Oncology, Inc. Anti-pd-1 antibodies and related binding molecules and methods and uses thereof
WO2025259871A1 (en) 2024-06-14 2025-12-18 Gilead Sciences, Inc. Anti-ccr8 antibodies and uses thereof
WO2026006767A1 (en) 2024-06-28 2026-01-02 Dispatch Biotherapeutics, Inc. Tethered il-9/il-9r and related engineered cells and methods
WO2026069312A1 (en) 2024-09-24 2026-04-02 Yeda Research And Development Co. Ltd. Immune cells expressing an inducible therapeutic agent and uses thereof
WO2026072752A1 (en) 2024-09-27 2026-04-02 Pharma Cinq, Llc Rod-derived cone viability factor fusion protein
WO2026117756A2 (en) 2024-11-27 2026-06-04 Dispatch Biotherapeutics, Inc. Systems and methods for expression of synthetic cancer antigens and cytokine receptors
WO2026161387A1 (en) 2025-01-21 2026-07-30 Bristol-Myers Squibb Company Cd3 binding molecules and related methods and uses
WO2026161391A2 (en) 2025-01-21 2026-07-30 Bristol-Myers Squibb Company Cd19 binding molecules including cd3- and cd19- bispecific t cell engagers and related methods and uses

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
AU7906691A (en) * 1990-05-23 1991-12-10 United States of America, as represented by the Secretary, U.S. Department of Commerce, The 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
CA2098483A1 (en) 1993-05-09
KR930703453A (ko) 1993-11-30
CA2098483C (en) 2000-03-14
AU657829B2 (en) 1995-03-23
DE69232983D1 (de) 2003-05-08
JP2003174896A (ja) 2003-06-24
ATE236263T1 (de) 2003-04-15
ES2192555T3 (es) 2003-10-16
AU3134093A (en) 1993-06-07
DK0566732T3 (da) 2003-07-21
DE69232983T2 (de) 2003-11-13
IL103677A0 (en) 1993-04-04
EP0566732A1 (en) 1993-10-27
EP0566732B1 (en) 2003-04-02
JPH06504680A (ja) 1994-06-02
US5252479A (en) 1993-10-12
WO1993009239A1 (en) 1993-05-13
PT101040B (pt) 2003-06-30

Similar Documents

Publication Publication Date Title
DK0566732T3 (da) Adeno-associerede virus-2-basalvektorer
ES2147553T3 (es) Vectores virales recombinantes para la expresion en unas celulas musculares.
NL189092C (nl) Hybride menselijke leukocytinterferons, dubbelstrengs dna dat hiervoor codeert, expressievector, transformant micro-organisme, werkwijzen ter bereiding daarvan en farmaceutisch preparaat.
DE69128893D1 (de) Genetische veränderung von endothelzellen
ATE301201T1 (de) Für die gentherapie verwendbare plasmide
ATE323169T1 (de) Plasmid zur verabreichung von nukleine säuren und verwendungsverfahren
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
EP1446489A4 (en) INTEGRATED MINI CELLS AS VECTORS FOR DNA TRANSFER AND GENE THERAPY IN VITRO AND IN VIVO
BR9914643A (pt) Vetores adeno-associados para expressão dofator viii por células alvo
CN110747211A (zh) 基于crispr的改变基因产物表达的方法及其应用
ATE243741T1 (de) Zufuhr von genprodukten mittels mesangium-zellen
DE69910809D1 (de) Verwendung von OmpA Enterobakterproteinen um zielgerichtet Antigen-Präsentierende Zellen anzusteuern
DK0870044T3 (da) Reguleret proteinekspression i stabilt transfekterede pattedyrsceller
IL131436A0 (en) Compositions for treatment of disorders involving programmed cell death
Evans et al. Gene therapy in orthopaedics
Eck The prospects for gene therapy
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
Mashko et al. Creation of artificial hybrid operons with partially overlapping genes to achieve an expression of heterologous genes in Escherichia coli cells
AU2002359572A8 (en) 15603, a human ion channel family member
Zhao Gene Transfer and Drug Delivery by Electronic Pulse Delivery: A Nonviral Delivery Approach
AU2002352068A1 (en) 2150, a human protein kinase family member and uses therefor

Legal Events

Date Code Title Description
BB1A Laying open of patent application

Effective date: 19931210

FG3A Patent granted, date of granting

Effective date: 20030505

MM3A Annulment or lapse

Effective date: 20041105

NF3A Restitutio in integrum

Effective date: 20050530

MM4A Annulment/lapse due to non-payment of fees, searched and examined patent

Free format text: LAPSE DUE TO NON-PAYMENT OF FEES

Effective date: 20061106