ZA201807401B - Modulatory polynucleotides - Google Patents

Modulatory polynucleotides

Info

Publication number
ZA201807401B
ZA201807401B ZA2018/07401A ZA201807401A ZA201807401B ZA 201807401 B ZA201807401 B ZA 201807401B ZA 2018/07401 A ZA2018/07401 A ZA 2018/07401A ZA 201807401 A ZA201807401 A ZA 201807401A ZA 201807401 B ZA201807401 B ZA 201807401B
Authority
ZA
South Africa
Prior art keywords
modulatory polynucleotides
modulatory
polynucleotides
Prior art date
Application number
ZA2018/07401A
Other languages
English (en)
Inventor
Dinah Wen-Yee Sah
Jinzhao Hou
Mathieu Nonnenmacher
Original Assignee
Voyager Therapeutics 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 Voyager Therapeutics Inc filed Critical Voyager Therapeutics Inc
Publication of ZA201807401B publication Critical patent/ZA201807401B/en

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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • 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
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/52—Methods for regulating/modulating their activity modulating the physical stability, e.g. GC-content
    • 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
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychology (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
ZA2018/07401A 2016-05-18 2018-11-05 Modulatory polynucleotides ZA201807401B (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662338137P 2016-05-18 2016-05-18
US201762485050P 2017-04-13 2017-04-13
PCT/US2017/033268 WO2017201248A1 (en) 2016-05-18 2017-05-18 Modulatory polynucleotides

Publications (1)

Publication Number Publication Date
ZA201807401B true ZA201807401B (en) 2020-12-23

Family

ID=60325551

Family Applications (1)

Application Number Title Priority Date Filing Date
ZA2018/07401A ZA201807401B (en) 2016-05-18 2018-11-05 Modulatory polynucleotides

Country Status (13)

Country Link
US (3) US10584337B2 (enExample)
EP (1) EP3458588B1 (enExample)
JP (3) JP7066635B2 (enExample)
KR (3) KR20240056729A (enExample)
CN (2) CN117904112A (enExample)
AU (2) AU2017267665C1 (enExample)
CA (2) CA3252099A1 (enExample)
IL (2) IL262784B2 (enExample)
MX (2) MX2018014154A (enExample)
RU (1) RU2758488C2 (enExample)
SG (1) SG11201809699XA (enExample)
WO (1) WO2017201248A1 (enExample)
ZA (1) ZA201807401B (enExample)

Families Citing this family (39)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112375760A (zh) * 2014-11-14 2021-02-19 沃雅戈治疗公司 调节性多核苷酸
KR20230169197A (ko) 2014-11-14 2023-12-15 보이저 테라퓨틱스, 인크. 근위축성 측삭 경화증(als)을 치료하는 조성물 및 방법
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
US10745447B2 (en) 2015-09-28 2020-08-18 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
IL297576B2 (en) 2016-05-18 2024-02-01 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
CN117904112A (zh) 2016-05-18 2024-04-19 沃雅戈治疗公司 调节性多核苷酸
CN119491003A (zh) 2017-05-05 2025-02-21 沃雅戈治疗公司 治疗亨廷顿病的组合物和方法
CA3061652A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
AU2018352236B2 (en) 2017-10-16 2025-04-10 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)
MX2020010464A (es) 2018-04-03 2021-01-29 Vectores de virus que evitan anticuerpos.
SG11202009451VA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
MX2020010465A (es) * 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
EP3807404A1 (en) 2018-06-13 2021-04-21 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
AU2019310459A1 (en) 2018-07-24 2021-02-18 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
TW202035689A (zh) 2018-10-04 2020-10-01 美商航海家醫療公司 測量病毒載體粒子的效價及強度之方法
JP2022512621A (ja) 2018-10-05 2022-02-07 ボイジャー セラピューティクス インコーポレイテッド Ａａｖ産生タンパク質をコードする操作された核酸コンストラクト
TW202028468A (zh) 2018-10-15 2020-08-01 美商航海家醫療公司 用於桿狀病毒/Sf9系統中rAAV之大規模生產的表現載體
WO2020150556A1 (en) 2019-01-18 2020-07-23 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
CN113924115A (zh) 2019-01-31 2022-01-11 俄勒冈健康与科学大学 用于aav衣壳的使用转录依赖性定向进化的方法
SG11202110146VA (en) 2019-03-21 2021-10-28 Stridebio Inc Recombinant adeno-associated virus vectors
MX2021013268A (es) * 2019-04-29 2021-11-17 Univ Pennsylvania Nuevas capsides de aav y composiciones que las contienen.
US20220243225A1 (en) 2019-04-29 2022-08-04 Voyager Therapeutics, Inc. SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS
US20220213503A1 (en) * 2019-05-02 2022-07-07 Boehringer Ingelheim International Gmbh Viral vectors and nucleic acids for use in the treatment of pf-ild and ipf
CN114072500B (zh) * 2019-08-02 2024-05-10 香港科技大学 控制microRNA表达的方法
US20220290182A1 (en) 2019-08-09 2022-09-15 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
US20220364114A1 (en) 2019-08-26 2022-11-17 Voyager Therapeutics, Inc. Controlled expression of viral proteins
CN114787180A (zh) 2019-10-17 2022-07-22 斯特里迪比奥公司 用于治疗c型尼曼-匹克病的腺相关病毒载体
EP4192514A1 (en) 2020-08-06 2023-06-14 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
TW202227632A (zh) 2020-08-19 2022-07-16 美商史崔德生物公司 用於治療雷特症候群之腺相關病毒載體
CA3210763A1 (en) 2021-02-12 2022-08-18 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
US20240141377A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2023214512A1 (ja) * 2022-05-06 2023-11-09 学校法人常翔学園 人工ｒｎａ分子
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
IL320511A (en) 2022-12-29 2025-06-01 Voyager Therapeutics Inc Compositions and methods for regulating MAPT
TW202449167A (zh) 2023-04-26 2024-12-16 美商航海家醫療公司 用於治療肌肉萎縮性脊髓側索硬化症之組成物及方法
CN120082552A (zh) * 2023-12-01 2025-06-03 艾码生物科技(南京)有限公司 初级-miRNA及其应用
WO2025122644A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt

Family Cites Families (516)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2640638B1 (fr) 1988-12-20 1991-02-15 Commissariat Energie Atomique Bioreacteur et dispositif pour la culture de cellules animales
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
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe 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
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
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
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
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
US5625048A (en) 1994-11-10 1997-04-29 The Regents Of The University Of California Modified green fluorescent proteins
CA2207927A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
US5652224A (en) 1995-02-24 1997-07-29 The Trustees Of The University Of Pennsylvania Methods and compositions for gene therapy for the treatment of defects in lipoprotein metabolism
US5741657A (en) 1995-03-20 1998-04-21 The Regents Of The University Of California Fluorogenic substrates for β-lactamase and methods of use
US6281010B1 (en) 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
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
US5688676A (en) 1995-06-07 1997-11-18 Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US6197293B1 (en) 1997-03-03 2001-03-06 Calydon, Inc. Adenovirus vectors specific for cells expressing androgen receptor and methods of use thereof
US6676935B2 (en) 1995-06-27 2004-01-13 Cell Genesys, Inc. Tissue specific adenoviral vectors
CA2230655C (en) 1995-08-30 2008-06-17 Genzyme Corporation Chromatographic purification of adenovirus and aav
US6265389B1 (en) 1995-08-31 2001-07-24 Alkermes Controlled Therapeutics, Inc. Microencapsulation and sustained release of oligonucleotides
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
US5952221A (en) 1996-03-06 1999-09-14 Avigen, Inc. Adeno-associated virus vectors comprising a first and second nucleic acid sequence
US7026468B2 (en) 1996-07-19 2006-04-11 Valentis, Inc. Process and equipment for plasmid purification
US6083716A (en) 1996-09-06 2000-07-04 The Trustees Of The University Of Pennsylvania Chimpanzee adenovirus vectors
WO1998010086A1 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania Methods using cre-lox for production of recombinant adeno-associated viruses
CA2264482A1 (en) 1996-09-06 1998-03-12 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
EP0932418B1 (en) 1996-09-06 2007-12-05 The Trustees Of The University Of Pennsylvania Method for recombinant adeno-associated virus-directed gene therapy
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
EP0932694A2 (en) 1996-09-11 1999-08-04 THE UNITED STATES GOVERNMENT as represented by THE DEPARTMENT OF HEALTH AND HUMAN SERVICES Aav4 vector and uses thereof
EP0968284B1 (en) 1996-11-20 2006-12-13 Introgen Therapeutics, Inc. An improved method for the production and purification of adenoviral vectors
US7732129B1 (en) 1998-12-01 2010-06-08 Crucell Holland B.V. Method for the production and purification of adenoviral vectors
AU741605B2 (en) 1996-12-18 2001-12-06 Targeted Genetics Corporation AAV split-packaging genes and cell lines comprising such genes for use in the production of recombinant AAV vectors
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6710036B2 (en) 1997-07-25 2004-03-23 Avigen, Inc. Induction of immune response to antigens expressed by recombinant adeno-associated virus
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
WO1999015677A1 (en) 1997-09-19 1999-04-01 The Trustees Of The University Of Pennsylvania Method for gene transfer using bcl2 and compositions useful therein
CA2304168A1 (en) 1997-09-19 1999-04-01 The Trustees Of The University Of Pennsylvania Methods and cell line useful for production of recombinant adeno-associated viruses
WO1999014354A1 (en) 1997-09-19 1999-03-25 The Trustees Of The University Of The Pennsylvania Methods and vector constructs useful for production of recombinant aav
US6642051B1 (en) 1997-10-21 2003-11-04 Targeted Genetics Corporation Amplifiable adeno-associated virus(AAV) packaging cassettes for the production of recombinant AAV vectors
IT1297074B1 (it) 1997-11-21 1999-08-03 Angeletti P Ist Richerche Bio Forme ormone-dipendenti delle proteine rep del virus adeno-associato (aav-2), sequenze di dna codificanti per esse, vettori che le
EP1042494A1 (en) 1997-12-23 2000-10-11 Introgene B.V. Adeno-associated virus and adenovirus chimeric recombinant viruses useful for the integration of foreign genetic information into the chromosomal dna of target cells
US6410300B1 (en) 1998-01-12 2002-06-25 The University Of North Carolina At Chapel Hill Methods and formulations for mediating adeno-associated virus (AAV) attachment and infection and methods for purifying AAV
WO1999043360A1 (en) 1998-02-26 1999-09-02 The Trustees Of The University Of Pennsylvania Stable protection from dystrophic sarcolemmal degeneration and restoration of the sarcoglycan complex
US6953690B1 (en) 1998-03-20 2005-10-11 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US6521426B1 (en) 1998-04-08 2003-02-18 Istituto Di Ricerche Di Biologia Molecolare P. Angeletti S.P.A. Preparation of recombinant adenovirus carrying a rep gene of adeno-associated virus
FR2778413B1 (fr) 1998-05-07 2000-08-04 Immunotech Sa Nouveaux reactifs et methode de lyse des erythrocytes
EP1078096A1 (en) 1998-05-11 2001-02-28 Ariad Gene Therapeutics, Inc. Multiviral compositions and uses thereof
US6436392B1 (en) 1998-05-20 2002-08-20 University Of Iowa Research Foundation Adeno-associated virus vectors
EP1849872A1 (en) 1998-05-20 2007-10-31 University Of Iowa Research Foundation Adeno-associated virus vectors and uses thereof
EP1080218A1 (en) 1998-05-27 2001-03-07 University of Florida Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient
CA2329143A1 (en) 1998-05-27 1999-12-02 Cell Genesys, Inc. Adeno-associated viral vector-mediated expression of factor viii activity
WO1999061601A2 (en) 1998-05-28 1999-12-02 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav5 vector and uses thereof
US6984517B1 (en) 1998-05-28 2006-01-10 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector and uses thereof
GB2338236B (en) 1998-06-13 2003-04-09 Aea Technology Plc Microbiological cell processing
US6900049B2 (en) 1998-09-10 2005-05-31 Cell Genesys, Inc. Adenovirus vectors containing cell status-specific response elements and methods of use thereof
US6416992B1 (en) 1998-10-13 2002-07-09 Avigen, Inc. Compositions and methods for producing recombinant adeno-associated virus
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
JP2002528087A (ja) 1998-10-27 2002-09-03 クルセル ホランド ベー ヴェー 改良ａａｖベクター産生
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
US6689600B1 (en) 1998-11-16 2004-02-10 Introgen Therapeutics, Inc. Formulation of adenovirus for gene therapy
US6759050B1 (en) 1998-12-03 2004-07-06 Avigen, Inc. Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
US6225113B1 (en) 1998-12-04 2001-05-01 Genvec, Inc. Use of trans-activation and cis-activation to modulate the persistence of expression of a transgene in an at least E4-deficient adenovirus
US6387368B1 (en) 1999-02-08 2002-05-14 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
DE19905501B4 (de) 1999-02-10 2005-05-19 MediGene AG, Gesellschaft für molekularbiologische Kardiologie und Onkologie Verfahren zur Herstellung eines rekombinanten Adeno-assoziierten Virus, geeignete Mittel hierzu sowie Verwendung zur Herstellung eines Arzneimittels
US6509150B1 (en) 1999-03-05 2003-01-21 Universite De Nantes Compositions and methods for recombinant Adeno-Associated Virus production
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
JP4693244B2 (ja) 1999-03-18 2011-06-01 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法
AU4807400A (en) 1999-04-30 2000-11-17 University Of Florida Adeno-associated virus-delivered ribozyme compositions and methods of use
WO2000075353A1 (en) 1999-06-02 2000-12-14 Trustees Of The University Of Pennsylvania Compositions and methods useful for production of recombinant viruses which require helper viruses
EP1190249A2 (en) 1999-06-08 2002-03-27 University Of Iowa Research Foundation Compounds and methods to enhance raav transduction
CN1378593A (zh) 1999-08-20 2002-11-06 约翰斯霍普金斯大学医学院 构建融合文库的方法和组合物及该文库的用途
EP1218035A2 (en) 1999-09-29 2002-07-03 The Trustees Of The University Of Pennsylvania Rapid peg-modification of viral vectors
US6365394B1 (en) 1999-09-29 2002-04-02 The Trustees Of The University Of Pennsylvania Cell lines and constructs useful in production of E1-deleted adenoviruses in absence of replication competent adenovirus
JP2003533170A (ja) 1999-10-07 2003-11-11 ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン アデノ随伴ウイルスおよびその使用
US7241447B1 (en) 1999-10-07 2007-07-10 University Of Iowa Research Foundation Adeno-associated virus vectors and uses thereof
WO2001032711A2 (en) 1999-10-21 2001-05-10 Board Of Trustees Of The University Of Arkansas Adeno-associated virus aav rep78 major regulatory protein, mutants thereof and uses thereof
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
IL149667A0 (en) 1999-12-10 2002-11-10 Ariad Gene Therapeutics Inc Un Methods for expression of genes in primates
US20020045264A1 (en) 2000-03-14 2002-04-18 During Matthew J. Production of chimeric capsid vectors
US7638120B2 (en) 2000-03-14 2009-12-29 Thomas Jefferson University High transgene expression of a pseudotyped adeno-associated virus type
US6468524B1 (en) 2000-03-22 2002-10-22 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services AAV4 vector and uses thereof
US6855314B1 (en) 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
US7048920B2 (en) 2000-03-24 2006-05-23 Cell Genesys, Inc. Recombinant oncolytic adenovirus for human melanoma
KR101215789B1 (ko) 2000-03-30 2012-12-26 화이트헤드 인스티튜트 포 바이오메디칼 리서치 Ｒｎａ 간섭의 ｒｎａ 서열 특이적인 매개체
GB0009887D0 (en) 2000-04-20 2000-06-07 Btg Int Ltd Cytotoxic agents
AU2001257611A1 (en) 2000-04-28 2001-11-12 Avigen, Inc. Polynucleotides for use in recombinant adeno-associated virus virion production
US20030013189A1 (en) 2000-04-28 2003-01-16 Wilson James M. Compositions and methods useful for non-invasive delivery of therapeutic molecules to the bloodstream
CA2406743A1 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
US20020106381A1 (en) 2000-06-13 2002-08-08 High Katherine A. Methods for administering recombinant adeno-associated virus virions to humans previously exposed to adeno-associated virus
DE60128916T2 (de) 2000-07-18 2007-09-20 Takeda Pharmaceutical Co. Ltd. Neues, physiologisch aktives peptid und dessen verwendung
US6593123B1 (en) 2000-08-07 2003-07-15 Avigen, Inc. Large-scale recombinant adeno-associated virus (rAAV) production and purification
US6329181B1 (en) 2000-08-07 2001-12-11 Neurologix, Inc. Helper functions for recombinant vector production
ATE366319T1 (de) 2000-08-17 2007-07-15 Keiya Ozawa Adeno-assoziierte virus-vermittelte übertragung von angiogenesefaktoren
DE10044384A1 (de) 2000-09-08 2002-04-18 Medigene Ag Wirtszellen zur Verpackung von rekombinantem Adeno-assoziiertem Virus (rAAV), Verfahren zu ihrer Herstellung und deren Verwendung
FR2813891B1 (fr) 2000-09-14 2005-01-14 Immunotech Sa Reactif multifonctionnel pour erythrocytes mettant en jeu des carbamates et applications
GB0024550D0 (enExample) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
JP2002153278A (ja) 2000-11-22 2002-05-28 Hisamitsu Pharmaceut Co Inc ウイルスベクターの製造に用いられる細胞、その製法およびその細胞を用いたウイルスベクターの製造方法
WO2002070719A2 (en) 2001-01-19 2002-09-12 Trustees Of The University Of Pennsylvania Regulatable gene expression system
FR2821624B1 (fr) 2001-03-01 2004-01-02 Sod Conseils Rech Applic Nouveau polynucleotide utilisable pour moduler la proliferation des cellules cancereuses
WO2002071843A1 (en) 2001-03-14 2002-09-19 Genteric, Inc. Recombinant adeno-assocaited virus-mediated gene transfer via retroductal infusion of virions
EP1379540A4 (en) * 2001-04-19 2005-11-16 Bristol Myers Squibb Co POLYNUCLEOTIDES AND POLYPEPTIDES ASSOCIATED WITH NF-KB PATH
US20040136963A1 (en) 2001-06-22 2004-07-15 The Trustees Of The University Of Pennsylvania Simian adenovirus vectors and methods of use
EP1409748B1 (en) 2001-06-22 2011-10-26 The Trustees of The University of Pennsylvania Recombinant Adenoviruses comprising simian adenovirus proteins and uses thereof.
EP1900815B1 (en) 2001-07-12 2016-09-07 University of Massachusetts In vivo production of small interfering RNAs that mediate gene silencing
ES2566561T3 (es) 2001-07-12 2016-04-13 University Of Massachusetts Producción in vivo de ARN pequeños de interferencia que median el silenciamiento génico
US8241622B2 (en) 2001-07-13 2012-08-14 University Of Iowa Research Foundation Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
EP1279740A1 (en) 2001-07-26 2003-01-29 Vrije Universiteit Brussel Recombinant vector derived from adeno-associated virus for gene therapy
WO2003014367A1 (en) 2001-08-08 2003-02-20 The Trustees Of The University Of Pennsylvania Method for purification of viral vectors having proteins which bind sialic acid
US20030092161A1 (en) 2001-09-19 2003-05-15 The Trustees Of The University Of Pennsylvania Compositions and methods for production of recombinant viruses, and uses therefor
EP2390328A1 (en) 2001-09-28 2011-11-30 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. MicroRNA molecules
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
EP1572893B1 (en) 2001-11-09 2009-01-07 THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of the DEPARTMENT OF HEALTH AND HUMAN SERVICES Production of adeno-associated virus in insect cells
KR101014207B1 (ko) 2001-11-13 2011-02-14 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 아데노-결합 바이러스 (ａａｖ) 서열을 검출 및/또는 확인하는 방법 및 그 방법에 의해 확인된 신규한 서열을 분리하는 방법
EP1944043A1 (en) 2001-11-21 2008-07-16 The Trustees of the University of Pennsylvania Simian adenovirus nucleic acid and amino acid sequences, vectors containing same, and methods of use
CA2852277C (en) 2001-11-21 2018-02-20 Guangping Gao Simian adenovirus nucleic acid and amino acid sequences, vectors containing same, and methods of use
EP1453537A1 (en) 2001-12-12 2004-09-08 FH Faulding & Co. Limited Composition for viral preservation
ES2526341T3 (es) 2001-12-17 2015-01-09 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 9 de virus adeno-asociado (AAV), vectores que las contienen, y usos de las mismas
CA2469785C (en) 2001-12-17 2014-04-22 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor
EP1463530A4 (en) 2001-12-19 2006-09-06 Lijun Wang ADENO-ASSOCIATED VIRUS-RELATED DELIVERY OF GDNF TO SKELETAL MUSCLES
CN1617938A (zh) 2002-01-16 2005-05-18 戴诺生物技术有限公司 从单个样品中分离核酸和蛋白质的方法
AU2003238456A1 (en) 2002-02-01 2003-09-02 Oxford Biomedica (Uk) Limited Viral vector
US20030180756A1 (en) 2002-03-21 2003-09-25 Yang Shi Compositions and methods for suppressing eukaryotic gene expression
GB0208390D0 (en) 2002-04-11 2002-05-22 Univ London Adeno-associated virus producer system
US20030198620A1 (en) 2002-04-16 2003-10-23 Keiya Ozawa Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions
ATE348153T1 (de) 2002-04-29 2007-01-15 Univ Pennsylvania Methode für die direkte gewinnung und amplifikation von integrierten viren aus zellulärer gewebe-dna
DK1501921T4 (da) 2002-04-30 2012-10-08 Oncolytics Biotech Inc Forbedrede virusrensningsmetoder
SI1496944T1 (sl) 2002-05-01 2009-02-28 Univ Florida Izboljšani sistemi ekspresije RAAV za genetsko modifikacijo specifičnih kapsidnih proteinov
SI1504126T1 (sl) 2002-05-03 2014-08-29 Duke University Office Of Science And Technology Postopek regulacije izraĹľanja genov
EP1783212B1 (en) 2002-05-14 2011-01-05 Merck Sharp & Dohme Corp. Methods of adenovirus purification
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
US20040241854A1 (en) 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
US20080274989A1 (en) 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
AU2003268323A1 (en) 2002-08-29 2004-03-19 The Board Of Trustees Of The Leland Stanford Junior University Circular nucleic acid vectors, and methods for making and using the same
US20040137471A1 (en) 2002-09-18 2004-07-15 Timothy Vickers Efficient reduction of target RNA's by single-and double-stranded oligomeric compounds
US7169612B2 (en) 2002-11-11 2007-01-30 Sanofi-Aventis Deutschland Gmbh Use of EDG2 receptor in an animal model of heart failure
EP1418185A1 (en) 2002-11-11 2004-05-12 Aventis Pharma Deutschland GmbH Use of EDG2 receptor in an animal model of heart failure
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
US7618948B2 (en) 2002-11-26 2009-11-17 Medtronic, Inc. Devices, systems and methods for improving and/or cognitive function through brain delivery of siRNA
US20080318210A1 (en) 2003-08-27 2008-12-25 Rosetta Genomics Bioinformatically detectable group of novel regulatory viral and viral associated oligonucleotides and uses thereof
JP2006523103A (ja) 2003-02-21 2006-10-12 ザ ペン ステイト リサーチ ファウンデーション Ｒｎａ干渉組成物および方法
US7510872B2 (en) 2003-02-26 2009-03-31 Nationwide Children's Hospital Recombinant adeno-associated virus production
US20070172460A1 (en) 2003-03-19 2007-07-26 Jurgen Kleinschmidt Random peptide library displayed on aav vectors
WO2004108922A2 (en) 2003-04-25 2004-12-16 The Trustees Of The University Of Pennsylvania Use of aav comprising a capsid protein from aav7 or aav8 for the delivery of genes encoding apoprotein a or e genes to the liver
AU2004239114B2 (en) 2003-05-14 2008-03-13 Japan Science And Technology Agency Inhibition of the expression of huntingtin gene
US8927269B2 (en) 2003-05-19 2015-01-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Avian adenoassociated virus and uses thereof
PT2277996E (pt) 2003-05-21 2014-12-16 Genzyme Corp Métodos para produzir preparações de vírions de aav recombinantes substancialmente livres de capsídeos vazios
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
JP4888876B2 (ja) 2003-06-13 2012-02-29 田平 武 アルツハイマー病の治療のための組換えアデノ随伴ウィルスベクター
PT2657247T (pt) 2003-06-19 2017-06-29 Genzyme Corp Viriões aav com imunorreatividade diminuída e seus usos
JP4754480B2 (ja) 2003-06-20 2011-08-24 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア キメラアデノウイルスの作成法およびそのようなキメラアデノウイルスの使用
US7291498B2 (en) 2003-06-20 2007-11-06 The Trustees Of The University Of Pennsylvania Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
ES2391975T3 (es) 2003-07-25 2012-12-03 Genvec, Inc. Vacunas a base de vector adenovírico
EP2530157B1 (en) 2003-07-31 2016-09-28 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of miRNAs
ES2808561T3 (es) 2003-09-12 2021-03-01 Univ Massachusetts Interferencia por ARN para el tratamiento de trastornos de ganancia de función
US20050064489A1 (en) 2003-09-24 2005-03-24 Zhang Fang Liang Engineered U6 and H1 promoters
EP2298926A1 (en) 2003-09-30 2011-03-23 The Trustees of The University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
JP4790619B2 (ja) 2003-10-27 2011-10-12 ロゼッタ インファーマティクス エルエルシー 遺伝子サイレンシングのためのｓｉＲＮＡを設計する方法
WO2005087926A2 (en) 2004-03-05 2005-09-22 Benitec, Inc. Multiple promoter expression cassettes for simultaneous delivery of rnai agents
DE602005026269D1 (de) 2004-04-28 2011-03-24 Univ Pennsylvania Sequenzielle freisetzung immunogener moleküle über adenoviren und adeno-assoziierte viren vermittelte abgaben
EP1751275B1 (en) 2004-06-01 2017-08-16 Avigen, Inc. Compositions and methods to prevent aav vector aggregation
EP2572661B1 (en) 2004-10-05 2019-11-20 Genzyme Corporation Stepped cannula
US7901921B2 (en) 2004-10-22 2011-03-08 Oncolytics Biotech Inc. Viral purification methods
WO2006055727A2 (en) 2004-11-18 2006-05-26 The Board Of Trustees Of The University Of Illinois MULTICISTRONIC siRNA CONSTRUCTS TO INHIBIT TUMORS
CN1286981C (zh) 2004-11-30 2006-11-29 华中科技大学同济医学院附属同济医院 表达人类cyp2j2反义基因的重组腺相关病毒及其制备方法
US20060229268A1 (en) 2004-12-16 2006-10-12 Alsgen, Llc Small interference RNA (siRNA) molecules for modulating superoxide dismutase (SOD)
CA2596711C (en) 2005-02-03 2015-12-15 Benitec, Inc. Rnai expression constructs
US8614101B2 (en) 2008-05-20 2013-12-24 Rapid Pathogen Screening, Inc. In situ lysis of cells in lateral flow immunoassays
US7625570B1 (en) 2005-03-10 2009-12-01 The Regents Of The University Of California Methods for purifying adeno-associated virus
US8999943B2 (en) 2005-03-14 2015-04-07 Board Of Regents, The University Of Texas System Antigene oligomers inhibit transcription
WO2006102072A2 (en) 2005-03-23 2006-09-28 The Trustees Of The University Of Pennsylvania Use of a pa131 polypeptide in treatment of atherosclerosis
CN101203613B (zh) 2005-04-07 2012-12-12 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
EP2420256B1 (en) 2005-05-02 2016-06-22 Genzyme Corporation Gene therapy for neurometabolic disorders
JP2009504782A (ja) 2005-08-18 2009-02-05 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 神経疾患を治療するための方法および組成物
AU2006283189B2 (en) 2005-08-23 2013-01-31 The Regents Of The University Of California Reflux resistant cannula and system for chronic delivery of therapeutic agents using convection-enhanced delivery
EP1857552A1 (en) 2006-05-20 2007-11-21 Cargill Incorporated Thermostable xylose isomerase enzyme
JP5305913B2 (ja) 2005-10-20 2013-10-02 アムステルダム モレキュラー セラピューティクス ビー．ブイ． 昆虫細胞で産生される改良されたａａｖベクター
RU2448974C2 (ru) * 2005-11-01 2012-04-27 Элнилэм Фармасьютикалз, Инк. РНКи-ИНГИБИРОВАНИЕ РЕПЛИКАЦИИ ВИРУСА ГРИППА
EP2431467A3 (en) 2005-11-17 2012-05-02 Board Of Regents, The University Of Texas Modulation of gene expression by oligomers targeted to chromosomal DNA
WO2007087451A2 (en) * 2006-01-25 2007-08-02 University Of Massachusetts Compositions and methods for enhancing discriminatory rna interference
US9150882B2 (en) 2006-01-31 2015-10-06 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
MX2008012219A (es) 2006-04-03 2008-10-02 Santaris Pharma As Composicion farmaceutica que comprende oligonucleotidos antisentido anti-miarn.
WO2008027084A2 (en) 2006-04-28 2008-03-06 The Trustees Of The University Of Pennsylvania Modified aav vectors having reduced capsid immunogenicity and use thereof
EP2018421B1 (en) 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Scalable production method for aav
DK2012822T3 (da) 2006-04-28 2010-05-25 Univ Pennsylvania Modificeret adenovirushexonprotein og anvendelser deraf
WO2007130519A2 (en) 2006-05-02 2007-11-15 Government Of The Usa, As Represented By The Secretary, Department Of Health And Human Services Viral nucleic acid microarray and method of use
WO2007139982A2 (en) 2006-05-25 2007-12-06 Sangamo Biosciences, Inc. Methods and compositions for gene inactivation
CA2655957C (en) 2006-06-21 2016-05-03 Amsterdam Molecular Therapeutics (Amt) B.V. Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells
JP5539718B2 (ja) 2006-07-21 2014-07-02 カリフォルニア インスティテュート オブ テクノロジー 樹状細胞の免疫付与用の、標的特異的な遺伝子輸送
US8945918B2 (en) 2006-08-24 2015-02-03 Virovek, Inc. Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor
HUE046709T2 (hu) 2006-10-03 2020-03-30 Genzyme Corp Génterápia amiotrófiás laterálszklerózis és más gerincvelõi rendellenességek kezelésére
WO2008067398A2 (en) 2006-11-29 2008-06-05 University Of Iowa Research Foundation Alternative export pathways for vector expressed rna interference
CA2693178C (en) 2006-11-29 2018-12-04 Nationwide Children's Hospital, Inc. Myostatin inhibition for enhancing muscle and/or improving muscle function
WO2008094516A2 (en) 2007-01-29 2008-08-07 City Of Hope Multi-targeting short interfering rnas
EP2114433B1 (en) 2007-02-02 2014-04-09 Biogen Idec MA Inc. Use of semaphorin 6a for promoting myelination and oligodendrocyte differentiation
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
US9611302B2 (en) 2007-04-09 2017-04-04 University Of Florida Research Foundation, Inc. High-transduction-efficiency RAAV vectors, compositions, and methods of use
EP3492596A1 (en) 2007-04-09 2019-06-05 University of Florida Research Foundation, Inc. Raav vector compositions having tyrosine-modified capsid proteins and methods for use
WO2008128251A1 (en) 2007-04-17 2008-10-23 The Children's Hospital Of Philadelphia Humanized viral vectors and methods of use thereof
WO2008134646A2 (en) 2007-04-26 2008-11-06 University Of Iowa Research Foundation Rna interference suppression of neurodegenerative diseases and methods of use thereof
EP2012122A1 (en) 2007-07-06 2009-01-07 Medigene AG Mutated parvovirus structural proteins as vaccines
EP2530152B1 (en) 2007-05-31 2017-12-27 University of Iowa Research Foundation Reduction of off-target RNA interference toxicity
ES2602610T3 (es) 2007-05-31 2017-02-21 Medigene Ag Proteína estructural mutada de un parvovirus
US8053186B2 (en) 2007-06-15 2011-11-08 The Ohio State University Research Foundation Oncogenic ALL-1 fusion proteins for targeting Drosha-mediated microRNA processing
US8841437B2 (en) * 2008-06-20 2014-09-23 The Board Of Trustees Of The Leland Stanford Junior University Precursor miRNA loop-modulated target regulation
JP5108942B2 (ja) 2007-06-29 2012-12-26 エフ．ホフマン−ラ ロシュ アーゲー プロモーター
US20110111481A1 (en) 2007-06-29 2011-05-12 Chiang Li ENABLING THE USE OF LONG dsRNA FOR GENE TARGETING IN MAMMALIAN AND OTHER SELECTED ANIMAL CELLS
EP2176283B1 (en) 2007-07-14 2016-11-02 University of Iowa Research Foundation Methods and compositions for treating brain diseases
EP2019143A1 (en) 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
SI3093345T1 (sl) 2007-07-26 2019-08-30 Uniqure Ip B.V. Bakulovirusni vektorji, ki vsebujejo ponavljajoča kodirna zaporedja z različnimi kodonskimi preferencami
ES2873350T3 (es) 2007-08-27 2021-11-03 1Globe Health Inst Llc Composiciones de ARN interferente asimétrico y usos de las mismas
US8420372B2 (en) 2007-09-04 2013-04-16 Alexander Bello Porcine adeno-associated viruses
JP2010538675A (ja) 2007-09-19 2010-12-16 アムステルダム モレキュラー セラピューティクス ビー．ブイ． タンパク質産生の改善のためのａａｖ複製機構の使用
EP2205746A4 (en) 2007-10-04 2010-12-22 Univ Texas MODULATION OF GENE EXPRESSION WITH AGRNA AND GAPS WITH ANTISENSE TRANSCRIPTS AS A TARGET
EP2186283A4 (en) 2007-10-18 2011-03-09 Lg Electronics Inc METHOD AND SYSTEM FOR TRANSMITTING AND RECEIVING SIGNALS
BRPI0819774A2 (pt) 2007-11-28 2014-10-14 Univ Pennsylvania Subfamília c de adenovírus sadv-40, -31 e -34 de símio e seus usos
US8685387B2 (en) 2007-11-28 2014-04-01 The Trustees Of The University Of Pennsylvania Simian E adenoviruses SAdV-39, -25.2, -26, -30, -37, and -38
CN101883857B (zh) 2007-11-28 2015-08-12 宾夕法尼亚大学托管会 猿猴亚家族B腺病毒SAdV-28、-27、-29、-32、-33和-35及其应用
JP2011507554A (ja) * 2007-12-28 2011-03-10 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 遺伝子発現を増加させるための方法および組成物
CA2710398A1 (en) 2008-01-18 2009-10-15 Genentech, Inc. Methods and compositions for targeting polyubiquitin
DK2242840T3 (da) 2008-01-29 2019-10-21 Applied Genetic Tech Corporation Produktion af rekombinant adeno-associeret virus under anvendelse af bhk-celler i suspension
CA2715289C (en) 2008-02-11 2019-12-24 Rxi Pharmaceuticals Corporation Modified rnai polynucleotides and uses thereof
ES2644880T3 (es) 2008-02-19 2017-11-30 Uniqure Ip B.V. Optimización de expresión de proteínas Rep y Cap parvovirales en las células de insecto
WO2009136977A2 (en) 2008-03-04 2009-11-12 The Trustees Of The University Of Pennsylvania Simian adenoviruses sadv-36,-42.1, -42.2, and -44 and uses thereof
WO2009137006A2 (en) 2008-04-30 2009-11-12 The University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
WO2009134681A2 (en) 2008-04-30 2009-11-05 The Trustees Of The University Of Pennsylvania Aav7 viral vectors for targeted delivery of rpe cells
AU2009274482A1 (en) 2008-05-20 2010-01-28 Eos Neuroscience, Inc. Vectors for delivery of light-sensitive proteins and methods of use
US9095126B2 (en) 2008-05-27 2015-08-04 Yale University Targeting TGF-β as a therapy for Alzheimer's disease
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
WO2009149182A1 (en) 2008-06-04 2009-12-10 The Board Of Regents Of The University Of Texas System Modulation of gene expression through endogenous small rna targeting of gene promoters
WO2009151620A2 (en) 2008-06-13 2009-12-17 Cornell Research Foundation, Inc. Wmc Pain treatment using erk2 inhibitors
US20110171262A1 (en) 2008-06-17 2011-07-14 Andrew Christian Bakker Parvoviral capsid with incorporated gly-ala repeat region
US8945885B2 (en) 2008-07-03 2015-02-03 The Board Of Trustees Of The Leland Stanford Junior University Minicircle DNA vector preparations and methods of making and using the same
US8212019B2 (en) 2008-07-30 2012-07-03 University Of Massachusetts Nucleic acid silencing sequences
KR20110086553A (ko) 2008-09-29 2011-07-28 암스테르담 몰레큘러 테라퓨틱스 비. 브이. 포르포빌리노겐 디아미나아제 유전자 치료
EP2340040B1 (en) 2008-10-22 2019-01-16 F.Hoffmann-La Roche Ag Modulation of axon degeneration
WO2010051367A1 (en) 2008-10-31 2010-05-06 The Trustees Of The University Of Pennsylvania Simian adenoviruses sadv-43, -45,-48,-49, and -50 and uses thereof
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
US8889641B2 (en) 2009-02-11 2014-11-18 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
US9464119B2 (en) 2009-03-04 2016-10-11 Deutsches Krebsforschungszentrum Assembly activating protein (AAP) and its use for the manufacture of parvovirus particles essentially consisting of VP3
MX2011010097A (es) 2009-03-27 2011-10-19 Proyecto Biomedicina Cima Sl Metodos y composiciones para el tratamiento de cirrosis y fibrosis hepatica.
CN102439157B (zh) 2009-04-30 2015-09-16 宾夕法尼亚大学托管会 包含腺伴随病毒构建体的靶向传导气道细胞组合物
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
EP2435575A2 (en) 2009-05-28 2012-04-04 Deutsches Krebsforschungszentrum Modified aav capsid polypeptides
CA2762203A1 (en) 2009-05-29 2010-12-02 Soumitra Roy Simian adenovirus 41 and uses thereof
EP2439274A4 (en) 2009-06-05 2013-11-06 Dai-Wu Seol MULTI-CISTRONIC shARN EXPRESSION CASSETTE FOR DELETING ONE OR MORE TARGET GENES
ES2693194T3 (es) 2009-06-16 2018-12-10 Genzyme Corporation Métodos mejorados para la purificación de vectores de AAV recombinantes
BR112012000421A2 (pt) 2009-07-06 2019-09-24 Alnylam Pharmaceuticals Inc composições e métodos para intensificar a produção de um produto biológico.
US20140004565A1 (en) 2009-07-06 2014-01-02 Alnylam Pharmaceuticals, Inc. Cell-based bioprocessing
ES2657737T3 (es) 2009-07-15 2018-03-06 Calimmune Inc. Vector dual para la inhibición del virus de la inmunodeficiencia humana
EP2292781A1 (en) 2009-08-17 2011-03-09 Genethon Baculovirus-based production of biopharmaceuticals free of contaminating baculoviral virions
WO2011038187A1 (en) 2009-09-25 2011-03-31 The Trustees Of The University Of Pennsylvania Controlled adeno-associated virus (aav) diversification and libraries prepared therefrom
CN102695526A (zh) 2009-11-09 2012-09-26 吉恩波多治疗股份公司 用于神经元特异性的体内连续dopa合成的新型病毒载体构建体
JP5697042B2 (ja) 2009-11-19 2015-04-08 国立大学法人 岡山大学 遺伝子発現を上昇させるシステム及び該システムを保持したベクター
WO2011069529A1 (en) 2009-12-09 2011-06-16 Curevac Gmbh Mannose-containing solution for lyophilization, transfection and/or injection of nucleic acids
ES2683695T3 (es) 2010-01-12 2018-09-27 The University Of North Carolina At Chapel Hill Repeticiones terminales invertidas restrictivas para vectores virales
ES2628889T3 (es) 2010-02-05 2017-08-04 The University Of North Carolina At Chapel Hill Composiciones y métodos para la transducción mejorada del parvovirus
US9163261B2 (en) 2010-02-22 2015-10-20 Koteswara Rao KOLLIPARA Adeno-associated virus 2/8—micro RNA-101 therapy for liver cancer
EP3792348A3 (en) 2010-03-11 2021-06-23 uniQure IP B.V. Mutated rep encoding sequences for use in aav production
EP2550021A2 (en) 2010-03-22 2013-01-30 Association Institut de Myologie Methods of increasing efficiency of vector penetration of target tissue
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
WO2011133890A1 (en) 2010-04-23 2011-10-27 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
WO2011133874A1 (en) 2010-04-23 2011-10-27 University Of Massachusetts Multicistronic expression constructs
EP2561075B1 (en) 2010-04-23 2018-06-27 University of Massachusetts Aav-based treatment of cholesterol-related disorders
US9839696B2 (en) 2010-04-30 2017-12-12 City Of Hope Recombinant adeno-associated vectors for targeted treatment
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
US8518907B2 (en) 2010-08-02 2013-08-27 Merck Sharp & Dohme Corp. RNA interference mediated inhibition of catenin (cadherin-associated protein), beta 1 (CTNNB1) gene expression using short interfering nucleic acid (siNA)
US8808684B2 (en) 2010-09-10 2014-08-19 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Epidermal growth factor receptor (EGFR) and methods of use in adenoviral-associated virus type 6 (AAV6) transduction
CN101972476B (zh) 2010-09-14 2012-12-19 中国人民解放军第二军医大学 利用MicroRNA-155的核酸疫苗佐剂及其构建方法
JP5010760B2 (ja) 2010-10-05 2012-08-29 タカラバイオ株式会社 ウイルスベクターの製造方法
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
CN107828820B (zh) 2010-10-27 2022-06-07 学校法人自治医科大学 用于向神经系统细胞导入基因的腺相关病毒粒子
US20130236433A1 (en) 2010-11-11 2013-09-12 Keith A. Webster Methods, compositions, cells, and kits for treating ischemic injury
EP3075860A1 (en) 2010-11-23 2016-10-05 The Trustees of the University of Pennsylvania Subfamily e simian adenovirus a1295 and uses thereof
CA2827380C (en) 2011-02-12 2020-08-25 University Of Iowa Research Foundation Therapeutic nucleic acids
SG10201601110VA (en) 2011-02-17 2016-03-30 Univ Pennsylvania Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer
GB201103062D0 (en) 2011-02-22 2011-04-06 Isis Innovation Method
EP2500434A1 (en) 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
EP3777843B1 (en) 2011-04-18 2023-01-04 National Center of Neurology and Psychiatry Drug delivery particle and method for producing the same
US20140031418A1 (en) 2011-04-20 2014-01-30 The Trustees Of The University Of Pennsylvania Regimens and Compositions for AAV-Mediated Passive Immunization of Airborne Pathogens
ES2661680T3 (es) 2011-04-21 2018-04-03 University Of Massachusetts Composiciones basadas en VAAr y métodos para tratar deficiencias de alfa-1 anti-tripsina
HRP20220036T1 (hr) 2011-04-22 2022-04-01 The Regents Of The University Of California Virioni adeno-povezanog virusa s varijantom kapsida i postupci njihove upotrebe
WO2012149646A1 (en) 2011-05-05 2012-11-08 Sunnybrook Research Institute Mirna inhibitors and their uses
US9249425B2 (en) 2011-05-16 2016-02-02 The Trustees Of The University Of Pennslyvania Proviral plasmids and production of recombinant adeno-associated virus
US9598468B2 (en) 2011-05-18 2017-03-21 University Of Florida Research Foundation, Incorporated Polypeptides and vectors for targeting HER2/neu expressing cells and uses thereof
CN103518132B (zh) 2011-06-06 2015-11-25 拜奥卡蒂斯股份有限公司 通过离子型表面活性剂选择性裂解细胞
EP3290055B1 (en) 2011-07-25 2024-08-28 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of dux4
ES2623780T3 (es) 2011-07-27 2017-07-12 Genethon Sistemas de expresión de baculovirus mejorados
US8852911B2 (en) 2011-08-04 2014-10-07 The Regents Of The University Of California Method of producing dicer
US20130129668A1 (en) * 2011-09-01 2013-05-23 The Regents Of The University Of California Diagnosis and treatment of arthritis using epigenetics
JP6198278B2 (ja) 2011-09-08 2017-09-20 ユニキュアー アイピー ビー．ブイ． Ａａｖ調製物からの混入ウイルスの除去
WO2013036889A1 (en) 2011-09-09 2013-03-14 University Of Washington Retrograde transport peptide and use of same for delivery to central nervous system
US9464322B2 (en) 2011-09-09 2016-10-11 University Of Kentucky Research Foundation Methods for diagnosing and treating alzheimer's disease (AD) using the molecules that stabilize intracellular calcium (Ca2+) release
CN109265543B (zh) 2011-09-19 2022-04-26 阿克松神经系统科学公司 阿尔茨海默病中的tau蛋白介导病变的基于蛋白质的疗法和诊断
CA2853482C (en) 2011-10-28 2020-05-19 Joshua Grieger Cell line for production of adeno-associated virus
WO2013078400A1 (en) 2011-11-22 2013-05-30 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
WO2013078199A2 (en) 2011-11-23 2013-05-30 Children's Medical Center Corporation Methods for enhanced in vivo delivery of synthetic, modified rnas
WO2013103896A1 (en) 2012-01-06 2013-07-11 Mayo Foundation For Medical Education And Research Treating cardiovascular or renal diseases
WO2013113696A1 (en) 2012-01-30 2013-08-08 Vib Vzw Means and method for diagnosis and treatment of alzheimer's disease
ES2660492T3 (es) 2012-02-14 2018-03-22 The Regents Of The University Of California Administración sistémica y expresión regulada de genes paracrinos para enfermedades cardiovasculares y otras afecciones
BR112014020325A2 (pt) 2012-02-17 2017-08-08 Childrens Hospital Philadelphia composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos
WO2013122605A1 (en) 2012-02-17 2013-08-22 Evernote Corporation Site memory processing
WO2013126605A1 (en) * 2012-02-21 2013-08-29 The Johns Hopkins University EXPRESSION CONSTRUCT FOR A LIN28-RESISTANT Let-7 PRECURSOR MICRORNA
RU2639277C2 (ru) 2012-02-29 2017-12-20 Сангамо Байосайенсиз, Инк. Способы и составы лечения болезни хантингтона
KR102063483B1 (ko) 2012-04-18 2020-01-08 더 칠드런스 호스피탈 오브 필라델피아 Ａａｖ 캡시드 변이체를 이용한 고효율 유전자 전달을 위한 조성물 및 방법
US20140162319A2 (en) 2012-05-02 2014-06-12 Sangeetha Hareendran Nucleotide sequences, methods, kit and a recombinant cell thereof
US9163259B2 (en) 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
AU2013259892B2 (en) 2012-05-08 2017-09-14 Alectos Therapeutics Inc. Permeable glycosidase inhibitors and uses thereof
EP3470523A1 (en) 2012-05-09 2019-04-17 Oregon Health & Science University Adeno associated virus plasmids and vectors
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
TWI698240B (zh) 2012-05-15 2020-07-11 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
EP2850194A4 (en) 2012-05-18 2016-06-08 Univ Pennsylvania APE-ADENOVIRES A1302, A1320, A1331 AND A1337 OF THE FAMILY E AND USES THEREOF
BR112014028666B1 (pt) 2012-05-18 2021-12-07 The General Hospital Corporation Uso de uma partícula de raav2, composição farmacêutica e kit
EP2852388A4 (en) 2012-05-23 2016-01-13 Univ Johns Hopkins COMPOUNDS AND METHOD FOR USE THEREOF FOR THE TREATMENT OF NEURODEEGENERATIVE DISEASES
KR101738438B1 (ko) 2012-07-06 2017-05-22 다카라 바이오 가부시키가이샤 아데노-관련 바이러스 벡터의 생산세포
CN104619832B (zh) 2012-07-06 2021-03-16 衣阿华大学研究基金会 经修饰的腺伴随病毒载体组合物
JP2015524264A (ja) 2012-07-17 2015-08-24 ユニベルシテ ドゥ ジュネーブ 遺伝子発現の下方制御のための核酸
BR112015002168A2 (pt) 2012-08-01 2017-11-07 Nationwide Childrens Hospital liberação intratecal de vírus 9 adeno-associado recombinante
JP2015529685A (ja) 2012-09-17 2015-10-08 ザ・リサーチ・インスティテュート・アット・ネイションワイド・チルドレンズ・ホスピタルＴｈｅ Ｒｅｓｅａｒｃｈ Ｉｎｓｔｉｔｕｔｅ Ａｔｎａｔｉｏｎｗｉｄｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ 筋萎縮性側索硬化症の処置のための組成物および方法
PL2900686T3 (pl) 2012-09-28 2021-01-25 The University Of North Carolina At Chapel Hill Wektory aav ukierunkowane na oligodendrocyty
AU2013204200B2 (en) 2012-10-11 2016-10-20 Brandeis University Treatment of amyotrophic lateral sclerosis
WO2014071042A1 (en) 2012-10-31 2014-05-08 The Trustees Of Columbia University In The City Of New York Methods for identifying candidates for the treatment of neurodegenerative diseases
RU2721275C2 (ru) 2012-12-12 2020-05-18 Те Брод Инститьют, Инк. Доставка, конструирование и оптимизация систем, способов и композиций для манипуляции с последовательностями и применения в терапии
EP2940131B1 (en) 2012-12-25 2019-02-20 Takara Bio Inc. Aav variant
US11299715B2 (en) 2013-01-08 2022-04-12 Genzyme Corporation Use of iNOS inhibitors to increase viral yield in culture
HK1218656A1 (zh) 2013-01-08 2017-03-03 贝尼泰克生物制药有限公司 年龄相关性黄斑变性治疗
US9066966B2 (en) 2013-02-01 2015-06-30 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia
ES2741502T3 (es) 2013-02-08 2020-02-11 Univ Pennsylvania Cápside de AAV8 modificada para la transferencia génica para terapias de retina
AU2014244167A1 (en) 2013-03-13 2015-10-08 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
US9447433B2 (en) 2013-03-15 2016-09-20 The University Of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
WO2014144844A1 (en) 2013-03-15 2014-09-18 The Board Of Trustees Of The Leland Stanford Junior University tRNA DERIVED SMALL RNAs (tsRNAs) INVOLVED IN CELL VIABILITY
JP2016517278A (ja) 2013-03-15 2016-06-16 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ スタッファー／フィラーポリヌクレオチド配列を含むベクターおよびその使用方法
CA2909085C (en) 2013-04-08 2023-08-29 University Of Iowa Research Foundation Chimeric adeno-associated virus/ bocavirus parvovirus vector
EP2792742A1 (en) 2013-04-17 2014-10-22 Universitätsklinikum Hamburg-Eppendorf (UKE) Gene-therapy vectors for treating cardiomyopathy
DK2986635T3 (en) 2013-04-18 2019-01-28 Fond Telethon EFFECTIVE DELIVERY OF BIG GENES THROUGH DUAL-AAV VECTORS
KR102268473B1 (ko) 2013-04-20 2021-06-25 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 엑손 2-표적 U7snRNA 폴리뉴클레오티드 작제물의 재조합형 아데노 부속 바이러스 전달
US9719106B2 (en) 2013-04-29 2017-08-01 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof
KR20240093939A (ko) 2013-05-15 2024-06-24 리젠츠 오브 더 유니버시티 오브 미네소타 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달
US10006049B2 (en) 2013-05-16 2018-06-26 University Of Florida Research Foundation, Incorporated Hairpin mRNA elements and methods for the regulation of protein translation
EA035322B1 (ru) * 2013-05-28 2020-05-28 ДиЭсБи-ЮЭсЭй ЛЛК Антитело с "запирающими" свойствами для инактивации лекарства белкового происхождения
CA2907799A1 (en) 2013-05-31 2014-12-04 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
WO2014201308A1 (en) 2013-06-12 2014-12-18 Washington University Endothelial-targeted adenoviral vectors, methods and uses therefor
US20160122727A1 (en) 2013-06-13 2016-05-05 Shire Human Genetic Therapies, Inc. Messenger rna based viral production
EP4012031A1 (en) * 2013-07-03 2022-06-15 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded rna
MX2016001044A (es) 2013-07-22 2016-09-09 The Children's Hospital Of Philadelphia Aav variante y composiciones, metodos y usos para transferencia genica a celulas, organos y tejidos.
MX373332B (es) 2013-07-26 2020-05-21 Univ Iowa Res Found UNA PARTÍCULA rAAV Y UN AGENTE DE INMUNOSUPRESIÓN PARA USARSE EN EL TRATAMIENTO DE ENFERMEDADES.
ITTO20130669A1 (it) 2013-08-05 2015-02-06 Consiglio Nazionale Ricerche Vettore adeno-associato ricombinante muscolo-specifico e suo impiego nel trattamento di patologie muscolari
LT3039146T (lt) 2013-08-27 2020-07-27 Research Institute at Nationwide Children`s Hospital Produktai ir būdai, skirti amiotropinės lateralinės sklerozės gydymui
US11078464B2 (en) 2013-08-30 2021-08-03 Amgen Inc. High titer recombinant AAV vector production in adherent and suspension cells
EP3561062A1 (en) 2013-09-13 2019-10-30 California Institute of Technology Selective recovery
US20160369298A1 (en) 2013-09-26 2016-12-22 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
EP3068889B1 (en) 2013-09-26 2019-04-17 Universitat Autònoma De Barcelona Gene therapy compositions for use in the prevention and/or treatment of non-alcoholic fatty liver disease
CN115141260B (zh) 2013-10-11 2025-05-20 马萨诸塞眼科耳科诊所 预测祖先病毒序列的方法及其用途
CN105745326A (zh) 2013-10-24 2016-07-06 优尼科Ip有限公司 用于基因治疗神经疾病的aav-5假型载体
WO2015069647A1 (en) 2013-11-05 2015-05-14 The Research Institute At Nationwide Children's Hospital COMPOSITIONS AND METHODS FOR INHIBITING NF- kB AND SOD-1 TO TREAT AMYOTROPHIC LATERAL SCLEROSIS
WO2015108610A1 (en) 2013-11-08 2015-07-23 The Board Of Trustees Of The University Of Arkansas Adeno-associated virus "x" oncogene
EP3068905A4 (en) 2013-11-11 2017-07-05 Sangamo BioSciences, Inc. Methods and compositions for treating huntington's disease
ES2690643T3 (es) 2013-11-26 2018-11-21 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Vectores virales adeno-asociados para el tratamiento de enfermedad de almacenamiento de glucógeno
CN105765066B (zh) 2013-11-29 2018-12-14 宝生物工程株式会社 用于定量腺伴随病毒的方法
WO2015084254A1 (en) 2013-12-03 2015-06-11 Agency For Science, Technology And Research Tailed Mirtron Effectors For RNAi-Mediated Gene Silencing
EP3080269B1 (en) 2013-12-09 2019-05-22 Baylor College of Medicine Hippo and dystrophin complex signaling in cardiomyocyte renewal
EP3080257A1 (en) 2013-12-12 2016-10-19 The Broad Institute Inc. Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
GB201322798D0 (en) 2013-12-20 2014-02-05 Oxford Biomedica Ltd Production system
WO2015106273A2 (en) 2014-01-13 2015-07-16 Trustees Of Boston University Methods and assays relating to huntingtons disease and parkinson's disease
GB201401707D0 (en) 2014-01-31 2014-03-19 Sec Dep For Health The Adeno-associated viral vectors
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
JP6640099B2 (ja) 2014-02-19 2020-02-05 エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ 血液脳関門シャトル
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
WO2015124546A1 (en) 2014-02-19 2015-08-27 Fundación Centro Nacional De Investigaciones Cardiovasculares Carlos Iii- Cnic Aav vectors for the treatment of ischemic and non-ischemic heart disease
WO2015126972A1 (en) 2014-02-21 2015-08-27 University Of Florida Research Foundation, Inc. Methods and compositions for gene delivery to on bipolar cells
JP6870988B2 (ja) 2014-02-24 2021-05-19 セルジーン コーポレイション 神経細胞増幅及び中枢神経系障害治療のためのセレブロンの活性化因子の使用方法
CA2941640A1 (en) 2014-03-04 2015-09-11 University Of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
AU2015226911B2 (en) 2014-03-07 2018-03-01 The Arizona Board Of Regents On Behalf Of The University Of Arizona Non-narcotic CRMP2 peptides targeting sodium channels for chronic pain
AU2015230094B2 (en) 2014-03-10 2021-05-27 Uniqure Ip B.V. Further improved AAV vectors produced in insect cells
US10280418B2 (en) 2014-03-18 2019-05-07 Univeristy Of Massachusetts RAAV-based compositions and methods for treating amyotrophic lateral sclerosis
EP4137573A3 (en) 2014-04-01 2023-05-03 Biogen MA Inc. Compositions for modulating sod-1 expression
WO2015157070A2 (en) 2014-04-09 2015-10-15 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating cystic fibrosis
EP2933335A1 (en) 2014-04-18 2015-10-21 Genethon A method of treating peripheral neuropathies and motor neuron diseases
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
US11555059B2 (en) 2014-04-25 2023-01-17 The Trustees Of The University Of Pennsylvania LDLR variants and their use in compositions for reducing cholesterol levels
CN106413760B (zh) 2014-05-08 2020-01-14 桑格摩生物科学股份有限公司 用于治疗亨廷顿病的方法和组合物
EP3142706A1 (en) 2014-05-16 2017-03-22 Vrije Universiteit Brussel Genetic correction of myotonic dystrophy type 1
RU2711147C2 (ru) 2014-05-20 2020-01-15 Юниверсити Оф Айова Рисерч Фаундейшн Терапевтические соединения для лечения болезни хантингтона
WO2015183667A1 (en) 2014-05-28 2015-12-03 The Regents Of The University Of California HYBRID tRNA/pre-miRNA MOLECULES AND METHODS OF USE
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
US20150376612A1 (en) * 2014-06-10 2015-12-31 The General Hospital Corporation CCCTC-Binding Factor (CTCF) RNA Interactome
EP3154560B1 (en) 2014-06-13 2019-03-13 University of Pittsburgh - Of the Commonwealth System of Higher Education Methods and materials for increasing viral vector infectivity
US10781459B2 (en) 2014-06-20 2020-09-22 University Of Florida Research Foundation, Incorporated Methods of packaging multiple adeno-associated virus vectors
WO2016004319A1 (en) 2014-07-02 2016-01-07 University Of Florida Research Foundation, Inc. Compositions and methods for purifying recombinant adeno-associated virus
WO2016006658A1 (ja) 2014-07-10 2016-01-14 タカラバイオ株式会社 非エンベロープウイルス粒子の製造方法
US9616090B2 (en) 2014-07-30 2017-04-11 Sangamo Biosciences, Inc. Gene correction of SCID-related genes in hematopoietic stem and progenitor cells
WO2016016449A1 (en) 2014-07-31 2016-02-04 Association Institut De Myologie Treatment of amyotrophic lateral sclerosis
WO2016019364A1 (en) 2014-08-01 2016-02-04 The Trustees Of The University Of Pennsylvania Compositions and methods for self-regulated inducible gene expression
EP3191593A4 (en) 2014-09-08 2018-01-24 University of Iowa Research Foundation Microrna inhibitor system and methods of use thereof
AU2015320694B2 (en) 2014-09-24 2021-11-11 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
JP6842410B2 (ja) 2014-10-03 2021-03-17 ユニバーシティ オブ マサチューセッツ 新規の高効率ライブラリーにより同定されるａａｖベクター
WO2016057975A2 (en) 2014-10-10 2016-04-14 Research Institute At Nationwide Children's Hospital Guided injections for aav gene transfer to muscle
AU2015335923B2 (en) 2014-10-21 2021-04-29 University Of Massachusetts Recombinant AAV variants and uses thereof
SG10202007103TA (en) 2014-11-05 2020-09-29 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
US10907130B2 (en) 2014-11-05 2021-02-02 Research Institute At Nationwide Children's Hospital Methods and materials for producing recombinant viruses in eukaryotic microalgae
KR20230169197A (ko) 2014-11-14 2023-12-15 보이저 테라퓨틱스, 인크. 근위축성 측삭 경화증(als)을 치료하는 조성물 및 방법
CN112375760A (zh) 2014-11-14 2021-02-19 沃雅戈治疗公司 调节性多核苷酸
WO2016081927A2 (en) 2014-11-21 2016-05-26 University Of Florida Research Foundation, Inc. Genome-modified recombinant adeno-associated virus vectors
AU2015349759B2 (en) 2014-11-21 2022-01-06 The University Of North Carolina At Chapel Hill Aav vectors targeted to the central nervous system
EP3591070B1 (en) 2014-11-28 2022-12-21 uniQure IP B.V. Dna impurities in a composition comprising a parvoviral virion
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
HUE043842T2 (hu) 2014-12-24 2019-09-30 Uniqure Ip Bv RNAi-indukált huntingtin gén szuppresszió
EP3240577B1 (en) 2014-12-30 2023-07-05 University of Iowa Research Foundation Therapeutic agent that activates mtorc1 function for use in treating huntington's disease
EP3245220B1 (en) 2015-01-14 2023-09-20 The University of North Carolina at Chapel Hill Methods and compositions for targeted gene transfer
CA2970730A1 (en) 2015-01-16 2016-07-21 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
JP2018506530A (ja) 2015-01-30 2018-03-08 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 脊柱軟膜下遺伝子送達システム
US20180030096A1 (en) 2015-02-03 2018-02-01 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
HUE057272T2 (hu) 2015-02-10 2022-04-28 Genzyme Corp Vírusrészecskék fokozott bevitele a csíkolt testbe és az agykéregbe
EA201791805A1 (ru) 2015-02-10 2018-05-31 Джензим Корпорейшн ВАРИАНТ СРЕДСТВА ДЛЯ RNAi
EP3262162A4 (en) 2015-02-23 2018-08-08 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
US20180094280A1 (en) 2015-03-20 2018-04-05 Bluebird Bio, Inc. Vector formulations
JP6836999B2 (ja) 2015-03-24 2021-03-03 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアＴｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ アデノ随伴ウイルス変異体及びその使用方法
CA2980337A1 (en) 2015-04-03 2016-10-06 University Of Massachusetts Oligonucleotide compounds for targeting huntingtin mrna
EP3929293A3 (en) 2015-04-03 2022-03-16 University Of Massachusetts Fully stabilized asymmetric sirna
US10081659B2 (en) 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
EP3283126B1 (en) 2015-04-16 2019-11-06 Emory University Recombinant promoters and vectors for protein expression in liver and use thereof
CA3019315A1 (en) 2015-04-23 2016-10-27 University Of Massachusetts Modulation of aav vector transgene expression
WO2016172008A1 (en) 2015-04-24 2016-10-27 University Of Massachusetts Modified aav constructions and uses thereof
WO2016179038A1 (en) 2015-05-01 2016-11-10 Spark Therapeutics, Inc. ADENO-ASSOCIATED VIRUS-MEDIATED CRISPR-Cas9 TREATMENT OF OCULAR DISEASE
HK1252512A1 (zh) 2015-05-07 2019-05-31 Massachusetts Eye & Ear Infirmary 向眼部遞送藥劑的方法
JP6993234B2 (ja) 2015-05-11 2022-01-13 アルキオーネ・ライフサイエンシズ・インコーポレイテッド 薬物送達システムおよび方法
EP3294894B8 (en) 2015-05-12 2019-09-25 The U.S.A. as represented by the Secretary, Department of Health and Human Services Aav isolate and fusion protein comprising nerve growth factor signal peptide and parathyroid hormone
US20170067028A1 (en) 2015-05-15 2017-03-09 Douglas J. Ballon Radiolabeling of adeno associated virus
WO2016191418A1 (en) 2015-05-26 2016-12-01 Salk Institute For Biological Studies Motor neuron-specific expression vectors
US11027024B2 (en) 2015-05-29 2021-06-08 University Of Iowa Research Foundation Methods of delivery of transgenes for treating brain diseases
RU2021134920A (ru) 2015-05-29 2022-04-15 Дзе Трастиз Оф Дзе Юнивёрсити Оф Пенсильвания Композиции и способы деградации неправильно упакованных белков
US11266748B2 (en) 2015-07-02 2022-03-08 University Of Florida Research Foundation, Incorporated Recombinant adeno-associated virus vectors to target medullary thyroid carcinoma
US10801040B2 (en) 2015-07-07 2020-10-13 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the peripheral nervous system of a subject
US20170007669A1 (en) 2015-07-07 2017-01-12 Mayo Foundation For Medical Education And Research Peptide-mediated delivery of active agents across the blood-brain barrier
WO2017015102A1 (en) 2015-07-17 2017-01-26 The Trustees Of The University Of Pennsylvania Compositions and methods for achieving high levels of transduction in human liver cells
US20180214576A1 (en) 2015-07-28 2018-08-02 University Of Massachusetts Transgenic expression of dnasei in vivo delivered by an adeno-associated virus vector
NZ778484A (en) 2015-07-30 2024-11-29 Massachusetts Eye & Ear Infirmary Ancestral virus sequences and uses thereof
WO2017023724A1 (en) 2015-07-31 2017-02-09 Voyager Therapeutics, Inc. Compositions and methods for the treatment of aadc deficiency
US20180201937A1 (en) 2015-08-04 2018-07-19 The University Of Chicago Inhibitors of cacna1a/alpha1a subunit internal ribosomal entry site (ires) and methods of treating spinocerebellar ataxia type 6
US10047377B2 (en) 2015-09-22 2018-08-14 Loyola University Of Chicago Methods for modulating KLHL1 levels, methods for modulating current activity in T-type calcium channels, molecules therefor, and methods for identifying molecules therefor
CN108348576B (zh) 2015-09-23 2022-01-11 桑格摩生物治疗股份有限公司 Htt阻抑物及其用途
US10745447B2 (en) 2015-09-28 2020-08-18 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
WO2017062983A1 (en) 2015-10-09 2017-04-13 The Children's Hospital Of Philadelphia Compositions and methods for treating huntington's disease and related disorders
CN108368505B (zh) 2015-10-09 2023-02-21 建新公司 改善的flare(流式细胞术减弱报道蛋白表达)技术用于快速批量分选
US10123969B2 (en) 2015-10-15 2018-11-13 Wisconsin Alumni Research Foundation Osmotic enhancement of drug/therapeutic delivery to the brain following infusion or injection into the cerebrospinal fluid
ES2978086T3 (es) 2015-10-22 2024-09-05 Univ Massachusetts Terapia génica con aspartoacilasa en el tratamiento de enfermedad de Canavan
WO2017070476A2 (en) 2015-10-22 2017-04-27 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav3 capsid library
CA3002980A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
WO2017070678A1 (en) 2015-10-23 2017-04-27 University Of Iowa Research Foundation Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection
US20180230489A1 (en) 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
MX2018005353A (es) 2015-10-28 2018-08-14 Univ Pennsylvania Administracion intratecal de vectores virales adenop-asociados para terapia genica.
WO2017079768A1 (en) 2015-11-08 2017-05-11 Genentech, Inc. Methods of screening for multispecific antibodies
US10633662B2 (en) 2015-11-10 2020-04-28 The Board Of Trustees Of The Leland Stanford Junior University Methods and compositions for modulating AAV infection
WO2017095753A1 (en) 2015-12-01 2017-06-08 Invivo Therapeutics Corporation Compositions and methods for preparing an injectable medium for administration into the central nervous system
PH12018501168B1 (en) 2015-12-01 2024-05-17 Spark Therapeutics Inc Scalable methods for producing recombinant adeno-associated viral (aav) vector in serum-free suspension cell culture system suitable for clinical use
US10406244B2 (en) 2015-12-02 2019-09-10 The Board Of Trustees Of The Leland Stanford Junior University AAV vectors with expanded packaging capacity
WO2017096164A1 (en) 2015-12-02 2017-06-08 The Board Of Trustees Of The Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
US11371061B2 (en) 2015-12-03 2022-06-28 Genethon Compositions and methods for improving viral vector efficiency
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
ES2918998T3 (es) 2015-12-11 2022-07-21 Univ Pennsylvania Método de purificación escalable para AAVrh10
US11015174B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV8
SG11201804713VA (en) 2015-12-11 2018-07-30 California Inst Of Techn TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)
US11098286B2 (en) 2015-12-11 2021-08-24 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV9
EP4659816A3 (en) 2015-12-14 2025-12-31 The University of North Carolina at Chapel Hill MODIFIED CAPSID PROTEINS FOR IMPROVED PARVOVIRUS VECTOR DELIVERY
WO2017112948A1 (en) 2015-12-24 2017-06-29 University Of Florida Research Foundation, Inc. Improved aav production using suspension adapted cells
JP6924441B2 (ja) 2016-01-15 2021-08-25 学校法人自治医科大学 てんかん治療のためのアデノ随伴ウイルスビリオン
US11826433B2 (en) 2016-02-02 2023-11-28 University Of Massachusetts Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system
US12070510B2 (en) 2016-02-05 2024-08-27 Emory University Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid
US11702672B2 (en) 2016-02-08 2023-07-18 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
US10179176B2 (en) 2016-02-16 2019-01-15 The Board Of Trustees Of The Leland Stanford Junior University Recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies
US20190071681A1 (en) 2016-02-26 2019-03-07 University Of Florida Research Foundation, Incorporated Aav heparin mutants that display significantly improved eye and brain transduction
KR102336362B1 (ko) 2016-03-03 2021-12-08 보이저 테라퓨틱스, 인크. 비-바이러스 유전자 전달을 위한 폐쇄형-말단 선형 듀플렉스 dna
CA3198936A1 (en) 2016-03-07 2017-09-14 University Of Iowa Research Foundation Aav-mediated expression using a synthetic promoter and enhancer
EP3429605A4 (en) 2016-03-18 2019-09-18 The Children's Hospital of Philadelphia THERAPEUTIC FOR THE TREATMENT OF DISEASES INCLUDING THE CENTRAL NERVOUS SYSTEM
WO2017165859A1 (en) 2016-03-24 2017-09-28 Research Institute At Nationwide Children's Hospital Modified viral capsid proteins
WO2017172733A1 (en) 2016-03-28 2017-10-05 The Regents Of The University Of California Anti-ryk antibodies and methods of using the same
US11013774B2 (en) 2016-03-28 2021-05-25 Ultragenyx Pharmaceutical Inc. Methods of heat inactivation of adenovirus
BR112018070250A2 (pt) 2016-03-30 2019-01-29 Spark Therapeutics Inc linhagem celular para proteína recombinante e/ou produção de vetores virais
PE20190434A1 (es) 2016-03-31 2019-03-21 Spark Therapeutics Inc PROCESO DE PRODUCCION DE rAAV A BASE DE COLUMNAS COMPLETAMENTE ESCALABLE
EP3443108A4 (en) 2016-04-15 2019-11-20 The Trustees of The University of Pennsylvania NOVEL AAV8 MUTANT CAPSIDES AND COMPOSITIONS THEREOF
BR112018071180A2 (pt) 2016-04-16 2019-02-12 University Of Florida Research Foundation, Incorporated métodos de reforço potência biológica de baculovirus system-produzido vírus adeno-associado recombinante
KR20230079511A (ko) 2016-04-21 2023-06-07 바이로베크 인코포레이티드 곤충 세포에서의 aav 생산, 및 이를 위한 방법 및 조성물
EP3235516B1 (en) 2016-04-22 2019-06-26 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts Universitätsmedizin Regulatable adeno-associated virus (aav) vector
US10585103B2 (en) 2016-04-28 2020-03-10 The Trustees Of Indiana University Methods and compositions for resolving components of a virus preparation
US20190224339A1 (en) 2016-04-29 2019-07-25 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3452495B1 (en) 2016-05-03 2021-06-23 Children's Medical Research Institute Adeno-associated virus polynucleotides, polypeptides and virions
US11866462B2 (en) 2016-05-04 2024-01-09 Oregon Health & Science University Recombinant adeno-associated viral vectors
IL297576B2 (en) 2016-05-18 2024-02-01 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
CN117904112A (zh) * 2016-05-18 2024-04-19 沃雅戈治疗公司 调节性多核苷酸
US10898585B2 (en) 2017-04-14 2021-01-26 Ptc Therapeutics .Inc. Gene therapy for AADC deficiency
TWI897844B (zh) 2017-05-05 2025-09-21 美商航海家醫療公司 調節多核苷酸
CN119491003A (zh) 2017-05-05 2025-02-21 沃雅戈治疗公司 治疗亨廷顿病的组合物和方法
CA3061652A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
US20220090127A1 (en) 2017-06-02 2022-03-24 Institut National De La Sante Et De La Recherche Medicale (Inserm Viral vector combining gene therapy and genome editing approaches for gene therapy of genetic disorders
GB201714027D0 (en) 2017-09-01 2017-10-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of huntington's disease
AU2018352236B2 (en) 2017-10-16 2025-04-10 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)
JP7565218B2 (ja) 2018-07-02 2024-10-10 ボイジャー セラピューティクス インコーポレイテッド 筋萎縮性側索硬化症および脊髄に関連する障害の治療
CN113286895A (zh) * 2018-09-05 2021-08-20 阿莫内塔诊断股份公司 用于诊断和治疗脑疾患，尤其是认知障碍的长的非编码RNA(lncRNA)

Also Published As

Publication number Publication date
IL262784B2 (en) 2023-10-01
RU2018140495A3 (enExample) 2020-10-14
KR102652994B1 (ko) 2024-04-01
BR112018073384A2 (pt) 2019-03-06
US11193129B2 (en) 2021-12-07
CA3024448A1 (en) 2017-11-23
JP2019519218A (ja) 2019-07-11
WO2017201248A1 (en) 2017-11-23
MX2018014154A (es) 2019-05-06
IL302748A (en) 2023-07-01
RU2758488C2 (ru) 2021-10-28
SG11201809699XA (en) 2018-12-28
KR20190005886A (ko) 2019-01-16
KR20220054727A (ko) 2022-05-03
JP2023182824A (ja) 2023-12-26
KR20240056729A (ko) 2024-04-30
US20190169616A1 (en) 2019-06-06
AU2023203585B2 (en) 2025-09-04
JP2022106853A (ja) 2022-07-20
CN117904112A (zh) 2024-04-19
CN110214187B (zh) 2024-01-30
CA3252099A1 (en) 2025-06-05
EP3458588A1 (en) 2019-03-27
US10584337B2 (en) 2020-03-10
US20200149045A1 (en) 2020-05-14
CN110214187A (zh) 2019-09-06
IL262784A (en) 2018-12-31
KR102392236B1 (ko) 2022-05-03
US12084659B2 (en) 2024-09-10
AU2017267665B2 (en) 2023-03-16
CA3024448C (en) 2025-09-09
IL262784B1 (en) 2023-06-01
AU2017267665C1 (en) 2023-10-05
EP3458588A4 (en) 2020-01-15
JP7374254B2 (ja) 2023-11-06
EP3458588B1 (en) 2025-10-29
AU2017267665A1 (en) 2018-12-06
MX2023010042A (es) 2023-09-05
RU2018140495A (ru) 2020-06-18
AU2023203585A1 (en) 2023-09-07
JP7066635B2 (ja) 2022-05-13
IL302748B1 (en) 2025-09-01
US20220162609A1 (en) 2022-05-26
IL302748B2 (en) 2026-01-01

Similar Documents

Publication Publication Date Title
IL302748B2 (en) Modulatory polynucleotides
IL284949A (en) modulatory polynucleotides
DK3410992T3 (en) Tilbagetrækkeligt udløb til stomipose
AU201616677S (en) Cylindres
DK3770053T3 (en) Batterilås til en elcykel
PL3504506T3 (pl) Cel
GB201608521D0 (en) An actuation arrangement
GB201615693D0 (en) Combinations
AU201710580S (en) Bookrack
DK3481437T3 (en) Precursorer til radiofluorering
GB201704318D0 (en) Staubsauger
DK3430280T3 (en) Magnetisk leje til en turbomaskine
AU201713059S (en) SUNSHADE - square
GB201619778D0 (en) Polytunnel arrangement
HK40004410A (en) Modulatory polynucleotides
HK40020646A (en) Modulatory polynucleotides
GB2555582B (en) Covering
GB201712093D0 (en) Not known
AU5499P (en) T15-1218 Bituminaria bituminosa
GB201621210D0 (en) Polytunnel arrangement
AU201713060S (en) SUNSHADE - triangle
GB201602489D0 (en) Spectrometer
AU2016376V (en) NinbellaPurple Alyogyne huegelii
AU2016362V (en) JCU9 Desmanthus pernambucanus
AU2016359V (en) JCU6 Desmanthus bicornutus