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HK1222767A1 - 腺相關病毒介導基因轉移到中樞神經系統 - Google Patents

腺相關病毒介導基因轉移到中樞神經系統

Info

Publication number
HK1222767A1
HK1222767A1 HK16111030.1A HK16111030A HK1222767A1 HK 1222767 A1 HK1222767 A1 HK 1222767A1 HK 16111030 A HK16111030 A HK 16111030A HK 1222767 A1 HK1222767 A1 HK 1222767A1
Authority
HK
Hong Kong
Prior art keywords
adeno
nervous system
central nervous
gene transfer
associated virus
Prior art date
Application number
HK16111030.1A
Other languages
English (en)
Inventor
R Scott Mcivor
Lalitha R Belur
Walter Low
Carolyn Fairbanks
Karen Kozarsky
Original Assignee
Univ Minnesota
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 Univ Minnesota filed Critical Univ Minnesota
Publication of HK1222767A1 publication Critical patent/HK1222767A1/zh

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
    • A61K2039/507—Comprising a combination of two or more separate antibodies
    • 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
    • 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
    • C12N2750/14171—Demonstrated in vivo effect
    • 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
    • C12N2830/007—Vector systems having a special element relevant for transcription cell cycle specific enhancer/promoter combination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01076—L-Iduronidase (3.2.1.76)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Orthopedic Medicine & Surgery (AREA)
HK16111030.1A 2013-05-15 2016-09-20 腺相關病毒介導基因轉移到中樞神經系統 HK1222767A1 (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361823757P 2013-05-15 2013-05-15
PCT/US2014/038209 WO2014186579A1 (en) 2013-05-15 2014-05-15 Adeno-associated virus mediated gene transfer to the central nervous system

Publications (1)

Publication Number Publication Date
HK1222767A1 true HK1222767A1 (zh) 2017-07-14

Family

ID=51898870

Family Applications (2)

Application Number Title Priority Date Filing Date
HK16110439.0A HK1222093A1 (zh) 2013-05-15 2016-09-01 腺相關病毒介導的基因向中樞神經系統轉移
HK16111030.1A HK1222767A1 (zh) 2013-05-15 2016-09-20 腺相關病毒介導基因轉移到中樞神經系統

Family Applications Before (1)

Application Number Title Priority Date Filing Date
HK16110439.0A HK1222093A1 (zh) 2013-05-15 2016-09-01 腺相關病毒介導的基因向中樞神經系統轉移

Country Status (13)

Country Link
US (11) US9827295B2 (zh)
EP (2) EP2996475A4 (zh)
JP (4) JP2016523835A (zh)
KR (3) KR20220119187A (zh)
CN (3) CN115120745A (zh)
AU (3) AU2014265417B2 (zh)
BR (1) BR112015028605A8 (zh)
CA (2) CA3209821A1 (zh)
HK (2) HK1222093A1 (zh)
MX (2) MX2015015560A (zh)
RU (1) RU2692251C2 (zh)
SG (2) SG10201710448SA (zh)
WO (1) WO2014186579A1 (zh)

Families Citing this family (57)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11201507507PA (en) 2013-03-15 2015-10-29 Univ Pennsylvania Compositions and methods for treating mpsi
KR20220119187A (ko) 2013-05-15 2022-08-26 리젠츠 오브 더 유니버시티 오브 미네소타 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
SG10202007103TA (en) 2014-11-05 2020-09-29 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
ES2878451T3 (es) 2014-11-14 2021-11-18 Voyager Therapeutics Inc Polinucleótidos moduladores
BR112017010087A2 (pt) 2014-11-14 2018-06-05 Voyager Therapeutics, Inc. composições e métodos para tratar esclerose lateral amiotrófica (ela)
EP3230441A4 (en) 2014-12-12 2018-10-03 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
EP3245221A4 (en) * 2015-01-16 2018-06-13 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
CA3019315A1 (en) 2015-04-23 2016-10-27 University Of Massachusetts Modulation of aav vector transgene expression
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
EP4000631A1 (en) * 2015-05-15 2022-05-25 REGENXBIO Inc. Adeno-associated for therapeutic delivery to central nervous system
US11027024B2 (en) 2015-05-29 2021-06-08 University Of Iowa Research Foundation Methods of delivery of transgenes for treating brain diseases
KR20180067684A (ko) * 2015-10-23 2018-06-20 유니버시티 오브 아이오와 리써치 파운데이션 인지 보호를 제공하면서 질환 개시 및 진행을 지연시키기 위해 유전자 요법을 이용하는 신경퇴행성 질환의 치료 방법
RU2739384C2 (ru) * 2015-11-05 2020-12-23 Дзе Дженерал Хоспитал Корпорейшн Интратекальная доставка последовательностей нуклеиновых кислот, кодирующих abcd1, для лечения адреномиелоневропатии
CN109152847A (zh) * 2016-01-15 2019-01-04 桑格摩生物治疗股份有限公司 用于治疗神经疾病的方法和组合物
IL260959B2 (en) * 2016-02-03 2024-09-01 Univ Pennsylvania Gene therapy for the treatment of mucoid polysaccharomyces type I
WO2017147123A1 (en) * 2016-02-22 2017-08-31 The University Of North Carolina At Chapel Hill Aav-idua vector for treatment of mps i-associated blindness
US11185555B2 (en) 2016-04-11 2021-11-30 Noah James Harrison Method to kill pathogenic microbes in a patient
KR20190008237A (ko) * 2016-04-15 2019-01-23 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 Ii형 점액다당류증의 치료를 위한 유전자 요법
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11951121B2 (en) 2016-05-18 2024-04-09 Voyager Therapeutics, Inc. Compositions and methods for 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
IL264872B1 (en) 2016-08-18 2024-10-01 Univ California CRISPR-CAS genome engineering using a modular AAV delivery system
EP3506817A4 (en) 2016-08-30 2020-07-22 The Regents of The University of California METHOD FOR BIOMEDICAL TARGETING AND RELEASE, AND DEVICES AND SYSTEMS FOR IMPLEMENTING THEM
JP7035033B2 (ja) 2016-09-28 2022-03-14 コーバー、インコーポレイテッド 治療用ＭＯＴＳ－ｃ関連ペプチド
WO2018144441A1 (en) * 2017-01-31 2018-08-09 Regenxbio Inc. Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)
WO2018156892A1 (en) 2017-02-23 2018-08-30 Adrx, Inc. Peptide inhibitors of transcription factor aggregation
EP3607073B1 (en) 2017-04-03 2024-06-05 Encoded Therapeutics, Inc. Tissue selective transgene expression
BR112019021569A2 (pt) * 2017-04-14 2020-05-12 Regenxbio Inc. Precursor de iduronato-2-sulfatase humana recombinante glicosilada (ids), método para tratar um sujeito humano diagnosticado com mucopolissacaridose tipo ii (mps ii)
CN111108198A (zh) 2017-05-05 2020-05-05 沃雅戈治疗公司 治疗亨廷顿病的组合物和方法
CN110913866A (zh) 2017-05-05 2020-03-24 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
IL271193B1 (en) 2017-06-07 2024-09-01 Regeneron Pharma Preparations and methods for internalizing enzymes
EP3634978A1 (en) 2017-06-07 2020-04-15 Adrx, Inc. Tau aggregation inhibitors
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
SG11201912631PA (en) 2017-07-06 2020-01-30 Univ Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
TWI722310B (zh) 2017-08-03 2021-03-21 美商航海家醫療公司 用於aav之遞送之組合物及方法
WO2019060662A1 (en) 2017-09-22 2019-03-28 The Trustees Of The University Of Pennsylvania GENE THERAPY FOR THE TREATMENT OF MUCOPOLYSACCHARIDOSE TYPE II
BR112020006671A2 (pt) 2017-10-03 2020-12-01 Prevail Therapeutics, Inc. terapias genéticas para distúrbios lisossômicos
EP3692158A4 (en) 2017-10-03 2021-08-04 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMIAL DISORDERS
KR102693318B1 (ko) 2017-10-03 2024-08-07 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
TWI804518B (zh) 2017-10-16 2023-06-11 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
EP4454654A2 (en) 2017-10-16 2024-10-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
CN111727259B (zh) 2017-12-01 2024-04-19 编码治疗公司 工程化的dna结合蛋白
SG11202006169PA (en) 2017-12-29 2020-07-29 Helixmith Co Ltd Adeno-associated virus (aav) vector having hybrid hgf gene introduced thereto
WO2019226832A1 (en) * 2018-05-22 2019-11-28 Jie Shen Gene therapy for alzheimer's disease
CA3114199A1 (en) 2018-09-26 2020-04-02 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
SG11202107761SA (en) 2019-01-28 2021-08-30 Cohbar Inc Therapeutic peptides
EP3952923A4 (en) * 2019-04-10 2023-10-18 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMAL DISEASES
BR112021020183A2 (pt) 2019-04-10 2022-03-03 Prevail Therapeutics Inc Terapias gênicas para distúrbios lisossomais
KR20220006527A (ko) 2019-04-10 2022-01-17 프리베일 테라퓨틱스, 인크. 리소좀 장애에 대한 유전자 요법
BR112021020157A2 (pt) * 2019-04-10 2021-12-14 Prevail Therapeutics Inc Terapias gênicas para transtornos lisossomais
EP4072595A1 (en) 2019-12-10 2022-10-19 Takeda Pharmaceutical Company Limited Adeno associated virus vectors for the treatment of hunter disease
US20230132582A1 (en) * 2020-03-11 2023-05-04 Shanghai Belief-Delivery Biomed Co., Ltd. Novel use of aspirin compound in increasing nucleic acid expression
US20230414659A1 (en) * 2020-10-30 2023-12-28 Immusoft Corporation Methods of administering genetically modified b cells for in vivo delivery of therapeutic agents
AU2022214429A1 (en) 2021-02-01 2023-09-14 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
WO2022170082A1 (en) * 2021-02-05 2022-08-11 Regents Of The University Of Minnesota Methods for preventing cardiac or skeletal defects in diseases including mucopolysaccharidoses

Family Cites Families (120)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US200901A (en) * 1878-03-05 Improvement in machines for compressing and purifying air
JPS5929648A (ja) 1982-08-10 1984-02-16 Mitsubishi Chem Ind Ltd ペプチド類
US5624898A (en) 1989-12-05 1997-04-29 Ramsey Foundation Method for administering neurologic agents to the brain
WO1991007947A1 (en) 1989-12-05 1991-06-13 Ramsey Foundation Neurologic agents for nasal administration to the brain
US6407061B1 (en) 1989-12-05 2002-06-18 Chiron Corporation Method for administering insulin-like growth factor to the brain
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
EP0733103B1 (en) 1993-11-09 2004-03-03 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
DK0728214T3 (da) 1993-11-09 2004-11-29 Targeted Genetics Corp Stabile cellelinjer, der er i stand til at udtrykke det adenoassocierede virusreplikationsgen
WO1996017947A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
US5656785A (en) 1995-08-07 1997-08-12 The Charles Stark Draper Laboratory, Inc. Micromechanical contact load force sensor for sensing magnitude and distribution of loads and tool employing micromechanical contact load force sensor
US6190659B1 (en) 1996-09-17 2001-02-20 The Rockefeller University Bacterial plasmin binding protein and methods of use thereof
EP1010228B1 (en) 1996-11-19 2007-02-28 Surgx Corporation A transient voltage protection device and method of making same
EP0950091A2 (en) 1996-12-18 1999-10-20 Targeted Genetics Corporation Aav split-packaging genes and cell lines comprising such genes for use in the production of recombinant aav vectors
AU8672198A (en) * 1997-07-31 1999-02-22 Chiron Corporation Method enabling readministration of aav vector via immunosuppression of host
EP1658857A1 (en) * 1997-10-29 2006-05-24 Genzyme Corporation Compositions and methods for treating lysosomal storage disease
WO1999061066A2 (en) 1998-05-27 1999-12-02 Avigen, Inc. Convection-enhanced delivery of aav vectors
US7273618B2 (en) 1998-12-09 2007-09-25 Chiron Corporation Method for administering agents to the central nervous system
DE69928563T2 (de) 1998-12-09 2006-07-27 Chiron Corp. (N.D.Ges.D. Staates Delaware), Emeryville Abgeben von neurotropischen Wirkstoffen an das Zentralnervensystem
AU2173400A (en) 1998-12-09 2000-06-26 Chiron Corporation Method for administering agents to the central nervous system
US20050032841A1 (en) * 1999-04-20 2005-02-10 Steven Walkley Therapeutic compositions and methods of treating glycolipid storage related disorders
US6569661B1 (en) 1999-11-12 2003-05-27 Biomarin Pharmaceutical Inc. Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
US6585971B1 (en) 1999-11-12 2003-07-01 Harbor-Ucla Research And Education Institute Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating disease caused by deficiencies thereof
WO2001036603A2 (en) * 1999-11-17 2001-05-25 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
JP2003516360A (ja) 1999-12-09 2003-05-13 カイロン コーポレイション 中枢神経系およびリンパ系に対するサイトカイン投与方法
US7084126B1 (en) 2000-05-01 2006-08-01 Healthpartners Research Foundation Methods and compositions for enhancing cellular function through protection of tissue components
US20040204379A1 (en) * 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
CA2416492C (en) 2000-07-18 2008-04-29 Duke University Treatment of glycogen storage disease type ii
US20020014242A1 (en) 2000-07-31 2002-02-07 Abraham Scaria Use of rapamycin to inhibit immune response and induce tolerance to gene therapy vector and encoded transgene products
AU2002211688A1 (en) 2000-10-13 2002-04-29 Chiron Corporation Method for treating ischemic events affecting the central nervous system
US20020169102A1 (en) 2001-04-03 2002-11-14 Frey William H. Intranasal delivery of agents for regulating development of implanted cells in the CNS
JP2004532849A (ja) 2001-04-20 2004-10-28 カイロン コーポレイション 中枢神経系へのポリヌクレオチド薬剤の送達
US20030229025A1 (en) 2002-02-25 2003-12-11 Chiron Corporation Intranasal administration of MC4-R agonists
US7485314B2 (en) 2002-05-06 2009-02-03 Los Angeles Biomedical Research Institute At Harbor-Ucla Medical Center Induction of antigen specific immunologic tolerance
AU2003302724A1 (en) * 2002-08-13 2004-07-09 Mcivor, Scott, R. Methods of using vectors to treat metabolic disorders
CA2499170A1 (en) 2002-09-27 2004-04-08 Children's Medical Center Corporation Methods and compositions for treatment of neurological disorder
US8512710B2 (en) 2002-11-22 2013-08-20 Ansun Biopharma, Inc. Class of therapeutic protein based molecules
US7446098B2 (en) * 2003-02-18 2008-11-04 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiencies
CA2500523A1 (en) 2003-05-01 2004-11-18 University Of Florida Research Foundation, Inc. Vp2-modified raav vector compositions and uses therefor
US20050026823A1 (en) 2003-06-20 2005-02-03 Biomarin Pharmaceutical Inc. Use of the chaperone receptor-associated protein (RAP) for the delivery of therapeutic compounds to the brain and other tissues
US7442372B2 (en) 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
US8071364B2 (en) * 2003-12-24 2011-12-06 Transgenrx, Inc. Gene therapy using transposon-based vectors
US7776312B2 (en) 2004-08-13 2010-08-17 Healthpartners Research Foundation Method of treating Alzheimer's disease comprising administering deferoxamine (DFO) to the upper one-third of the nasal cavity
US7618615B2 (en) 2004-08-13 2009-11-17 Healthpartners Research Foundation Methods for providing neuroprotection for the animal central nervous system against neurodegeneration caused by ischemia
US20090136505A1 (en) 2005-02-23 2009-05-28 Johanna Bentz Intranasal Administration of Active Agents to the Central Nervous System
EP1853288A2 (en) 2005-02-23 2007-11-14 Alza Corporation, Inc. Intranasal administration of active agents to the central nervous system
AU2006282799B2 (en) 2005-08-26 2012-11-29 Healthpartners Research & Education Methods for treatment of headaches by administration of oxytocin
HUE041928T2 (hu) * 2006-02-08 2019-06-28 Genzyme Corp Génterápia A-típusú Niemann-Pick-betegségre
US20100221235A1 (en) * 2006-02-08 2010-09-02 Diatos Compositions and Methods for Treating Lysosomal Storage Diseases
EP3459441A1 (en) * 2006-02-09 2019-03-27 Genzyme Corporation Slow intraventricular delivery
WO2007127163A2 (en) 2006-04-24 2007-11-08 Geron Corporation Cns-tumor treatment method and composition
EP1915986A1 (en) 2006-10-23 2008-04-30 BIOPHARM GESELLSCHAFT ZUR BIOTECHNOLOGISCHEN ENTWICKLUNG VON PHARMAKA mbH Lipid growth factor formulations
AU2008218109A1 (en) 2007-02-23 2008-08-28 University Of Florida Research Foundation, Inc. Compositions and methods for treating glycogen storage diseases
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
PT2158322T (pt) * 2007-06-06 2017-08-09 Genzyme Corp Terapia genética para doenças do armazenamento lisossomal
JP2011504163A (ja) 2007-06-08 2011-02-03 ヘルスパートナーズ リサーチ ファウンデーション 中枢神経系への治療化合物の標的化を高めるための薬学的組成物および方法
US8283160B2 (en) 2007-09-11 2012-10-09 Frey Ii William H Methods, pharmaceutical compositions and articles of manufacture for administering therapeutic cells to the animal central nervous system
EP2058401A1 (en) * 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
WO2009075815A1 (en) 2007-12-07 2009-06-18 Duke University Immunomodulating gene therapy
ES2751999T3 (es) 2008-01-29 2020-04-02 Applied Genetic Tech Corporation Producción recombinante de virus adeno-asociados usando células BHK en suspensión
US9265843B2 (en) * 2008-03-27 2016-02-23 The Ohio State University Treatment of metabolic-related disorders using hypothalamic gene transfer of BDNF and compositions therefor
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
US9415121B2 (en) * 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
WO2010071832A1 (en) * 2008-12-19 2010-06-24 Nationwide Children's Hospital Delivery of polynucleotides across the blood brain barrier using recombinant aav9
US11219696B2 (en) * 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
US7989502B2 (en) 2009-02-06 2011-08-02 Sri International Intranasal delivery of modafinil
WO2010093784A2 (en) 2009-02-11 2010-08-19 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
US20110070241A1 (en) * 2009-06-30 2011-03-24 Duke University Methods for modulating immune responses to aav gene therapy vectors
US8622993B2 (en) 2009-12-18 2014-01-07 Healthpartners Research Foundation Device and method for delivering therapeutic substances to the maxillary sinus of a patient
ES2698203T3 (es) * 2010-04-23 2019-02-01 Univ Massachusetts Vectores de AAV que se dirigen al SNC y métodos de uso de los mismos
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
CN103260637B (zh) * 2010-06-25 2016-04-06 夏尔人类遗传性治疗公司 乙酰肝素n-硫酸酯酶cns递送的方法和组合物
KR102007044B1 (ko) * 2010-06-25 2019-08-02 샤이어 휴먼 지네틱 테라피즈 인크. 치료제들의 cns 전달
NZ702803A (en) * 2010-06-25 2017-05-26 Shire Human Genetic Therapies Cns delivery of therapeutic agents
DK2593131T3 (da) * 2010-06-25 2019-10-14 Shire Human Genetic Therapies Fremgangsmåder og sammensætninger til cns-levering af iduronat-2-sulfatase
CN103282046B (zh) * 2010-06-25 2015-05-13 夏尔人类遗传性治疗公司 芳基硫酸酯酶 a cns 递送的方法和组合物
AR082319A1 (es) * 2010-07-22 2012-11-28 Biomarin Pharm Inc Produccion de una n-acetilgalactosamina-6-sulfatasa humana activa altamente fosforilada y sus usos
US9409953B2 (en) 2011-02-10 2016-08-09 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
PT2691529T (pt) 2011-03-31 2019-09-27 Univ Iowa Res Found Partícula de aav2 que compreende uma proteína do capsídeo de aav2 e um vetor que compreende um ácido nucleico que codifica uma tripeptidil peptidase 1 (tpp1) para utilização no tratamento da lipofuscinose ceroide infantil tardia (lincl) num mamífero não roedor por injeção intraventricular ou entrega icv
ES2702496T3 (es) * 2011-04-21 2019-03-01 Nationwide Childrens Hospital Inc Productos de virus recombinante y procedimientos para la inhibición de la expresión de la miotilina
US10196636B2 (en) * 2011-04-21 2019-02-05 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of myotilin
US8609088B2 (en) * 2011-05-10 2013-12-17 Regents Of The University Of Minnesota Intranasal delivery of therapeutic enzymes to the central nervous system for the treatment of lysosomal storage diseases
US10227387B2 (en) * 2011-05-18 2019-03-12 Children's Hospital Medical Center Targeted delivery of proteins across the blood-brain barrier
US20130039888A1 (en) * 2011-06-08 2013-02-14 Nationwide Children's Hospital Inc. Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders
US9387236B2 (en) 2011-06-10 2016-07-12 Prothera Inc. Pharmaceutical compositions containing protease and methods for the treatment of lysosomal storage diseases
ES2648169T3 (es) * 2011-07-25 2017-12-28 Nationwide Children's Hospital, Inc. Productos de virus recombinantes y métodos para inhibición de la expresión de DUX4
JP6329483B2 (ja) * 2011-10-12 2018-05-23 シナジェバ・バイオファーマ・コーポレイションＳｙｎａｇｅｖａ Ｂｉｏｐｈａｒｍａ Ｃｏｒｐ． 組換えヒトｎａｇｌｕタンパク質およびその利用
US9821149B2 (en) 2011-10-14 2017-11-21 Healthpartners Research & Education Methods for using ultrasound for enhancing efficiency and targeting of intranasal administration of therapeutic compounds to the central nervous system
AU2012328682B2 (en) 2011-10-27 2017-09-21 Sangamo Therapeutics, Inc. Methods and compositions for modification of the HPRT locus
EP3711778B1 (en) * 2011-12-02 2024-05-08 Armagen, Inc. Methods and compositions for increasing arylsulfatase a activity in the cns
NZ627992A (en) 2012-02-07 2017-02-24 Global Bio Therapeutics Inc Compartmentalized method of nucleic acid delivery and compositions and uses thereof
EP2861263B1 (en) 2012-06-19 2017-12-06 University of Florida Research Foundation, Inc. Compositions and methods for treating diabetes
EP3536795A3 (en) 2012-06-21 2019-12-11 Association Institut de Myologie Widespread gene expression
DK2879719T3 (en) * 2012-08-01 2018-09-03 Nationwide Childrens Hospital INTRATEKAL ADMINISTRATION OF RECOMBINANT ADENOASSOCATED VIRUSES
US12024568B2 (en) * 2012-09-13 2024-07-02 Cornell University Treatment of brain cancers using central nervous system mediated gene transfer of monoclonal antibodies
ES2679374T3 (es) * 2012-11-27 2018-08-24 Biomarin Pharmaceutical Inc. Proteínas de fusión terapéuticas dirigidas de enzima lisosómica y usos de las mismas
US9862945B2 (en) * 2013-04-20 2018-01-09 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus delivery of exon 2-targeted U7SNRNA polynucleotide constructs
US10821154B2 (en) 2013-05-01 2020-11-03 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy
KR20220119187A (ko) * 2013-05-15 2022-08-26 리젠츠 오브 더 유니버시티 오브 미네소타 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달
WO2015013148A2 (en) 2013-07-26 2015-01-29 University Of Iowa Research Foundation Methods and compositions for treating brain diseases
CA2920261C (en) 2013-08-08 2018-04-03 Global Bio Therapeutics, Inc. Clamp device for minimally invasive procedures and uses thereof
CA2922500A1 (en) * 2013-08-27 2015-03-05 Research Institute At Nationwide Children's Hospital Products and methods for treatment of amyotrophic lateral sclerosis
EP3060669A1 (en) * 2013-10-24 2016-08-31 uniQure IP B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
EP3909602A1 (en) 2014-04-25 2021-11-17 University of Florida Research Foundation, Inc. Methods of permitting a subject to receive multiple doses of recombinant adeno-associated virus
CA2946593A1 (en) * 2014-05-02 2015-11-05 Genzyme Corporation Aav vectors for retinal and cns gene therapy
US10053671B2 (en) 2014-06-20 2018-08-21 Wisconsin Alumni Research Foundation (Warf) Mutations that confer genetic stability to additional genes in influenza viruses
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
AU2015364636B9 (en) 2014-12-16 2021-12-02 Board Of Regents Of The University Of Nebraska Gene therapy for Juvenile Batten Disease
US10538589B2 (en) 2015-01-14 2020-01-21 Armagen Inc. Methods and compositions for increasing N-acetylglucosaminidase (NAGLU) activity in the CNS using a fusion antibody comprising an anti-human insulin receptor antibody and NAGLU
EP4000631A1 (en) 2015-05-15 2022-05-25 REGENXBIO Inc. Adeno-associated for therapeutic delivery to central nervous system
EP3328399B1 (en) 2015-07-31 2023-12-27 Regents of the University of Minnesota Modified cells and methods of therapy
IL260959B2 (en) 2016-02-03 2024-09-01 Univ Pennsylvania Gene therapy for the treatment of mucoid polysaccharomyces type I
PL3411484T3 (pl) 2016-02-05 2024-02-19 Emory University Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego
US11338046B2 (en) * 2016-09-30 2022-05-24 Esteve Pharmaceuticals, S.A. Adenoassociated virus vectors for the treatment of mucopolysaccharidoses
EP3541946A1 (en) 2016-11-15 2019-09-25 Regents Of The University Of Minnesota Method for improving neurological function in mpsi and mpsii and other neurological disorders
EP3570670B1 (en) * 2017-01-17 2024-03-06 Children's Medical Center Corporation Compositions and methods for treating lysosomal storage diseases and disorders
EP3621612A4 (en) 2017-05-11 2021-06-16 The Trustees of The University of Pennsylvania GENE THERAPY OF NEURONAL CEROIDS-LIPOFUSCINOSES
SG11201912631PA (en) 2017-07-06 2020-01-30 Univ Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
WO2022170082A1 (en) 2021-02-05 2022-08-11 Regents Of The University Of Minnesota Methods for preventing cardiac or skeletal defects in diseases including mucopolysaccharidoses
WO2022221400A2 (en) * 2021-04-13 2022-10-20 Capsida, Inc. Aav compositions having high expression levels in brain

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