CA2998603C - Gene therapy for neurometabolic disorders - Google Patents

Gene therapy for neurometabolic disorders Download PDF

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Publication number
CA2998603C
CA2998603C CA2998603A CA2998603A CA2998603C CA 2998603 C CA2998603 C CA 2998603C CA 2998603 A CA2998603 A CA 2998603A CA 2998603 A CA2998603 A CA 2998603A CA 2998603 C CA2998603 C CA 2998603C
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CA
Canada
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composition according
asm
site
aav
disease
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Expired - Lifetime
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CA2998603A
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English (en)
French (fr)
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CA2998603A1 (en
Inventor
Marco A. Passini
James Dodge
Gregory R. Stewart
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Genzyme Corp
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Genzyme Corp
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Expired - Lifetime legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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
    • C12N2750/14041—Use of virus, viral particle or viral elements as a 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
    • C12N2750/14141—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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
CA2998603A 2005-05-02 2006-05-02 Gene therapy for neurometabolic disorders Expired - Lifetime CA2998603C (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US67705705P 2005-05-02 2005-05-02
US60/677057 2005-05-02
US68580805P 2005-05-31 2005-05-31
US60/685808 2005-05-31
CA2607173A CA2607173C (en) 2005-05-02 2006-05-02 Composition comprising an adeno-associated virus vector having a serotype 1 capsid for use in treating lysosomal storage diseases or alzheimer's disease

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
CA2607173A Division CA2607173C (en) 2005-05-02 2006-05-02 Composition comprising an adeno-associated virus vector having a serotype 1 capsid for use in treating lysosomal storage diseases or alzheimer's disease

Publications (2)

Publication Number Publication Date
CA2998603A1 CA2998603A1 (en) 2006-11-09
CA2998603C true CA2998603C (en) 2019-07-23

Family

ID=37308324

Family Applications (2)

Application Number Title Priority Date Filing Date
CA2998603A Expired - Lifetime CA2998603C (en) 2005-05-02 2006-05-02 Gene therapy for neurometabolic disorders
CA2607173A Expired - Lifetime CA2607173C (en) 2005-05-02 2006-05-02 Composition comprising an adeno-associated virus vector having a serotype 1 capsid for use in treating lysosomal storage diseases or alzheimer's disease

Family Applications After (1)

Application Number Title Priority Date Filing Date
CA2607173A Expired - Lifetime CA2607173C (en) 2005-05-02 2006-05-02 Composition comprising an adeno-associated virus vector having a serotype 1 capsid for use in treating lysosomal storage diseases or alzheimer's disease

Country Status (13)

Country Link
US (2) US10632213B2 (cg-RX-API-DMAC7.html)
EP (4) EP3520823B1 (cg-RX-API-DMAC7.html)
JP (3) JP5829372B2 (cg-RX-API-DMAC7.html)
CN (4) CN101212988A (cg-RX-API-DMAC7.html)
AT (1) ATE525092T1 (cg-RX-API-DMAC7.html)
AU (1) AU2006243776A1 (cg-RX-API-DMAC7.html)
BR (1) BRPI0611379A2 (cg-RX-API-DMAC7.html)
CA (2) CA2998603C (cg-RX-API-DMAC7.html)
ES (1) ES2887076T3 (cg-RX-API-DMAC7.html)
IL (2) IL187078A (cg-RX-API-DMAC7.html)
PL (4) PL3058959T3 (cg-RX-API-DMAC7.html)
PT (4) PT3520823T (cg-RX-API-DMAC7.html)
WO (1) WO2006119458A1 (cg-RX-API-DMAC7.html)

Families Citing this family (39)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP3302529B2 (ja) 1995-04-28 2002-07-15 株式会社クボタ ポンプ吸水槽の整流装置
CA2606490C (en) * 2005-05-02 2018-02-06 Genzyme Corporation Gene therapy for spinal cord disorders
JP5829372B2 (ja) 2005-05-02 2015-12-09 ジェンザイム・コーポレーション 神経代謝性疾患のための遺伝子治療
HUE031156T2 (en) 2006-06-07 2017-06-28 Genzyme Corp Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
ES2635726T3 (es) 2007-06-06 2017-10-04 Genzyme Corporation Terapia génica para enfermedades de almacenamiento lisosómico
US8735082B2 (en) 2008-11-10 2014-05-27 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Gene signature for predicting prognosis of patients with solid tumors
HRP20250897T1 (hr) 2009-05-02 2025-09-26 Genzyme Corporation Genska terapija za neurodegenerativne poremećaje
US9150926B2 (en) 2010-12-06 2015-10-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Diagnosis and treatment of adrenocortical tumors using human microRNA-483
US20130090374A1 (en) * 2011-04-20 2013-04-11 Miguel Sena-Esteves Methods for the treatment of tay-sachs disease, sandhoff disease, and gm1-gangliosidosis
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
RU2716991C2 (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
US11319555B2 (en) 2014-11-20 2022-05-03 Duke University Compositions, systems and methods for cell therapy
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
CN107530447A (zh) 2015-02-10 2018-01-02 建新公司 病毒颗粒至纹状体和皮质的增强递送
AU2016235685B2 (en) * 2015-03-26 2019-04-18 Shanghai Auzone Biological Technology Co., Ltd. Method of diagnosis or treatment of neurological disorders with p75ECD and/or p75
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
WO2016196507A1 (en) * 2015-05-29 2016-12-08 University Of Iowa Research Foundation Methods of delivery of transgenes for treating brain diseases
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
KR102427379B1 (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
WO2018044933A1 (en) 2016-08-30 2018-03-08 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
JP2020518258A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法
JP2020518259A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ハンチントン病治療組成物および方法
DK3635009T3 (da) 2017-06-07 2026-03-30 Regeneron Pharma Sammensætninger og fremgangsmåder til internalisering af enzymer
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
EP3808849A1 (en) 2017-08-03 2021-04-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
AU2018338728B2 (en) 2017-09-29 2025-01-02 Centre National De La Recherche Scientifique (Cnrs) Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery
EP3697908A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
US12258597B2 (en) 2018-02-07 2025-03-25 Regeneron Pharmaceuticals, Inc. Methods and compositions for therapeutic protein delivery
KR20210019996A (ko) 2018-05-15 2021-02-23 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 조성물 및 방법
BR112020023145A2 (pt) 2018-05-17 2021-02-02 Regeneron Pharmaceuticals, Inc. anticorpo anti-cd63 ou fragmento de ligação ao antígeno do mesmo, molécula de ligação ao antígeno biespecífica, proteína terapêutica de múltiplos domínios, polinucleotídeo composição farmacêutica, e, composto
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
KR20240126916A (ko) * 2023-02-14 2024-08-22 경북대학교 산학협력단 Asm 단백질 또는 이의 단편을 포함하는 퇴행성 신경질환 또는 우울증 예방 또는 치료용 조성물

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SE8303626D0 (sv) 1983-06-23 1983-06-23 Kabigen Ab A recombinant plasmid a transformant microorganism, a polydoxyrebonucleotide segment, a process for producing a biologically active protein, and the protein thus produced
US5672344A (en) 1987-12-30 1997-09-30 The Regents Of The University Of Michigan Viral-mediated gene transfer system
US5773278A (en) 1991-05-03 1998-06-30 Mount Sinai Medical Center Acid sphingomyelinase gene
EP0705344B8 (en) 1993-06-24 2006-05-10 Advec Inc. Adenovirus vectors for gene therapy
AU687117B2 (en) 1993-10-25 1998-02-19 Canji, Inc. Recombinant adenoviral vector and methods of use
WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
US7252989B1 (en) 1994-04-04 2007-08-07 Board Of Regents, The University Of Texas System Adenovirus supervector system
ATE386131T1 (de) 1994-04-13 2008-03-15 Univ Rockefeller Aav-vermittelte überbringung von dna in zellen des nervensystems
AU1525797A (en) 1996-04-22 1997-11-12 Medtronic, Inc. Two-stage angled venous cannula
US6544785B1 (en) 1998-09-14 2003-04-08 Mount Sinai School Of Medicine Of New York University Helper-free rescue of recombinant negative strand RNA viruses
US6582692B1 (en) * 1999-11-17 2003-06-24 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
US20030165481A1 (en) * 2000-02-24 2003-09-04 Hersh Louis B. Amyloid peptide inactivating enzyme to treat Alzheimer's disease
JP2004516016A (ja) 2000-09-18 2004-06-03 ジェンザイム・コーポレイション ハイブリッドユビキチンプロモーターを含む発現ベクター
US20030050273A1 (en) * 2001-08-29 2003-03-13 Keiya Ozawa Compositions and methods for treating neurodegenerative diseases
US7232670B2 (en) * 2001-09-28 2007-06-19 St. Jude Children's Research Hospital Targeting proteins to cells expressing mannose receptors via expression in insect cells
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
IL162630A0 (en) 2001-12-21 2005-11-20 Salk Inst For Biological Studi A composition containing a viral vector containinga heterologous gene to be transduced into a neuron
US6998118B2 (en) 2001-12-21 2006-02-14 The Salk Institute For Biological Studies Targeted retrograde gene delivery for neuronal protection
WO2003100031A2 (en) * 2002-05-20 2003-12-04 Board Of Regents, The University Of Texas System Methods and compositions for delivering enzymes and nucleic acid molecules to brain, bone, and other tissues
US20040258666A1 (en) * 2003-05-01 2004-12-23 Passini Marco A. Gene therapy for neurometabolic disorders
EP1620133B1 (en) * 2003-05-01 2015-12-09 Genzyme Corporation Gene therapy for neurometabolic disorders
US7740168B2 (en) 2003-08-18 2010-06-22 Visa U.S.A. Inc. Method and system for generating a dynamic verification value
AU2003272868A1 (en) 2003-10-15 2005-04-27 Agtc Gene Technology Company Ltd. Method for large-scale production, isolation, purification and the uses of multi-type recombinant adeno-associated virus vectors
EP1716870A1 (en) 2005-04-29 2006-11-02 Bracco Imaging S.p.A. MRI contrast agents endowed with concentration independent responsiveness
JP5829372B2 (ja) 2005-05-02 2015-12-09 ジェンザイム・コーポレーション 神経代謝性疾患のための遺伝子治療
CA2606490C (en) 2005-05-02 2018-02-06 Genzyme Corporation Gene therapy for spinal cord disorders
AR059371A1 (es) 2006-02-08 2008-03-26 Genzyme Corp Terapia genica para la enfermedad de niemann-pick tipo a
KR20140045411A (ko) 2011-05-16 2014-04-16 젠자임 코포레이션 Ｃｘｃｒ４ 길항제의 용도
TWI821227B (zh) 2017-12-22 2023-11-11 美商健臻公司 胺甲喋呤誘發的免疫耐受性之生物標記

Also Published As

Publication number Publication date
PT3058959T (pt) 2019-05-24
US10632213B2 (en) 2020-04-28
PT3520823T (pt) 2021-09-16
EP2420256A1 (en) 2012-02-22
PL3520823T3 (pl) 2021-11-22
PL3058959T3 (pl) 2019-07-31
ATE525092T1 (de) 2011-10-15
EP3058959B1 (en) 2019-01-30
JP6338560B2 (ja) 2018-06-06
PT2420256T (pt) 2016-09-13
CA2607173C (en) 2018-04-24
EP3520823B1 (en) 2021-06-30
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