JP5829372B2 - 神経代謝性疾患のための遺伝子治療 - Google Patents

神経代謝性疾患のための遺伝子治療 Download PDF

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JP5829372B2
JP5829372B2 JP2008510224A JP2008510224A JP5829372B2 JP 5829372 B2 JP5829372 B2 JP 5829372B2 JP 2008510224 A JP2008510224 A JP 2008510224A JP 2008510224 A JP2008510224 A JP 2008510224A JP 5829372 B2 JP5829372 B2 JP 5829372B2
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aav
asm
mice
aav2
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JP2008540445A5 (cg-RX-API-DMAC7.html
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マルコ・エイ・パッシーニ
ジェイムズ・ダッジ
グレゴリー・アール・スチュアート
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    • C12N2799/00—Uses of viruses
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    • 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

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JP2008510224A 2005-05-02 2006-05-02 神経代謝性疾患のための遺伝子治療 Active JP5829372B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US67705705P 2005-05-02 2005-05-02
US60/677,057 2005-05-02
US68580805P 2005-05-31 2005-05-31
US60/685,808 2005-05-31
PCT/US2006/017242 WO2006119458A1 (en) 2005-05-02 2006-05-02 Gene therapy for neurometabolic disorders

Related Child Applications (1)

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JP2013201138A Division JP2014037418A (ja) 2005-05-02 2013-09-27 神経代謝性疾患のための遺伝子治療

Publications (3)

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JP2008540445A JP2008540445A (ja) 2008-11-20
JP2008540445A5 JP2008540445A5 (cg-RX-API-DMAC7.html) 2009-07-09
JP5829372B2 true JP5829372B2 (ja) 2015-12-09

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JP2008510224A Active JP5829372B2 (ja) 2005-05-02 2006-05-02 神経代謝性疾患のための遺伝子治療
JP2013201138A Pending JP2014037418A (ja) 2005-05-02 2013-09-27 神経代謝性疾患のための遺伝子治療
JP2015197051A Active JP6338560B2 (ja) 2005-05-02 2015-10-02 神経代謝性疾患のための遺伝子治療

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JP2013201138A Pending JP2014037418A (ja) 2005-05-02 2013-09-27 神経代謝性疾患のための遺伝子治療
JP2015197051A Active JP6338560B2 (ja) 2005-05-02 2015-10-02 神経代謝性疾患のための遺伝子治療

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US (2) US10632213B2 (cg-RX-API-DMAC7.html)
EP (4) EP3058959B1 (cg-RX-API-DMAC7.html)
JP (3) JP5829372B2 (cg-RX-API-DMAC7.html)
CN (4) CN104306986A (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) CA2607173C (cg-RX-API-DMAC7.html)
ES (1) ES2887076T3 (cg-RX-API-DMAC7.html)
IL (2) IL187078A (cg-RX-API-DMAC7.html)
PL (4) PL1879624T3 (cg-RX-API-DMAC7.html)
PT (4) PT2420256T (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
JP5766389B2 (ja) * 2005-05-02 2015-08-19 ジェンザイム・コーポレーション 脊髄疾患の遺伝子療法
AU2006243776A1 (en) 2005-05-02 2006-11-09 Genzyme Corporation Gene therapy for neurometabolic disorders
PL2489733T3 (pl) 2006-06-07 2019-08-30 Genzyme Corporation Terapia genowa w stwardnieniu zanikowym bocznym i innych zaburzeniach rdzenia kręgowego
SI2158322T1 (sl) * 2007-06-06 2017-10-30 Genzyme Corporation Genska terapija za bolezni lizosomskega shranjevanja
EP2687609B1 (en) 2008-11-10 2017-01-04 The United States of America, as represented by The Secretary, Department of Health and Human Services Method for treating solid tumor
BRPI1010868A2 (pt) 2009-05-02 2018-06-12 Genzyme Corp terapia gênica para distúrbios neurodegenerativos
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
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
DK3218386T3 (da) 2014-11-14 2021-06-07 Voyager Therapeutics Inc Modulatorisk polynukleotid
CA3193811A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
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
PL3256594T3 (pl) 2015-02-10 2022-02-14 Genzyme Corporation Ulepszone dostarczanie cząstek wirusa do prążkowia i kory
CN106794222B (zh) * 2015-03-26 2021-08-24 苏州澳宗生物科技有限公司 使用p75ecd和/或p75诊断或治疗神经障碍的方法
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
US11027024B2 (en) 2015-05-29 2021-06-08 University Of Iowa Research Foundation Methods of delivery of transgenes for treating brain diseases
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
WO2017189964A2 (en) 2016-04-29 2017-11-02 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
AU2017267665C1 (en) 2016-05-18 2023-10-05 Voyager Therapeutics, Inc. Modulatory polynucleotides
CN110650673B (zh) 2016-08-30 2024-04-09 加利福尼亚大学董事会 用于生物医学靶向和递送的方法以及用于实践该方法的装置和系统
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
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
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
JP7348844B2 (ja) 2017-06-07 2023-09-21 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 内部移行酵素のための組成物および方法
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
WO2019018342A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. NETWORK EQUIPMENT TRACK 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
US20200263199A1 (en) 2017-09-29 2020-08-20 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF 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)
SG11202007363TA (en) 2018-02-07 2020-08-28 Regeneron Pharma Methods and compositions for therapeutic protein delivery
US12319929B2 (en) 2018-05-15 2025-06-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of Parkinson's disease
US12252544B2 (en) 2018-05-17 2025-03-18 Regeneron Pharmaceuticals, Inc. Anti-CD63 antibodies, conjugates, and uses thereof
CA3114621A1 (en) 2018-09-28 2020-04-02 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
DE69434486T2 (de) 1993-06-24 2006-07-06 Advec Inc. Adenovirus vektoren für gentherapie
BR9407956A (pt) 1993-10-25 1996-11-26 Canji Inc Composiçao farmacêutica vetor de express o de adenovirus recombinante e kit par reduzir a proliferaçao de células tumorais
ES2216005T3 (es) 1993-11-09 2004-10-16 Targeted Genetics Corporation Produccion de titulos elevados de vectores de aav recombinantes.
US7252989B1 (en) 1994-04-04 2007-08-07 Board Of Regents, The University Of Texas System Adenovirus supervector system
CA2187626C (en) 1994-04-13 2009-11-03 Michael G. Kaplitt Aav-mediated delivery of dna to cells of the nervous system
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
AU2001290984A1 (en) 2000-09-18 2002-04-02 Genzyme Corporation Expression vectors containing hybrid ubiquitin promoters
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
EP2359869B1 (en) 2001-12-17 2018-12-26 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same and uses therefor
US6998118B2 (en) 2001-12-21 2006-02-14 The Salk Institute For Biological Studies Targeted retrograde gene delivery for neuronal protection
EP1478236B1 (en) 2001-12-21 2009-02-11 The Salk Institute For Biological Studies Targeted retrograde gene delivery to motor neurons
AU2003220115A1 (en) * 2002-05-20 2003-12-12 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
WO2005035743A1 (en) * 2003-10-15 2005-04-21 Vector 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
JP5766389B2 (ja) 2005-05-02 2015-08-19 ジェンザイム・コーポレーション 脊髄疾患の遺伝子療法
AU2006243776A1 (en) 2005-05-02 2006-11-09 Genzyme Corporation Gene therapy for neurometabolic disorders
WO2007092563A2 (en) 2006-02-08 2007-08-16 Genzyme Corporation Gene therapy for niemann-pick disease type a
JP2014513727A (ja) 2011-05-16 2014-06-05 ジェンザイム・コーポレーション Ｃｘｃｒ４拮抗薬の使用
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

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EP3058959A1 (en) 2016-08-24
EP3520823B1 (en) 2021-06-30
PL3058959T3 (pl) 2019-07-31
EP1879624B1 (en) 2011-09-21
JP2016041706A (ja) 2016-03-31
IL187078A (en) 2015-06-30
PL1879624T3 (pl) 2012-02-29
EP3058959B1 (en) 2019-01-30
CA2607173A1 (en) 2006-11-09
ATE525092T1 (de) 2011-10-15
EP1879624A4 (en) 2009-03-25
PT1879624E (pt) 2012-02-02
EP2420256A1 (en) 2012-02-22
WO2006119458A9 (en) 2007-02-15
US20140356327A9 (en) 2014-12-04
IL187078A0 (en) 2008-02-09
ES2887076T3 (es) 2021-12-21
JP2008540445A (ja) 2008-11-20
IL239167A0 (en) 2015-07-30
EP2420256B1 (en) 2016-06-22
PT3058959T (pt) 2019-05-24
US20200306387A1 (en) 2020-10-01
PL3520823T3 (pl) 2021-11-22
CN107362371A (zh) 2017-11-21
US10632213B2 (en) 2020-04-28
PT3520823T (pt) 2021-09-16
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JP2014037418A (ja) 2014-02-27
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CA2607173C (en) 2018-04-24
CA2998603C (en) 2019-07-23
AU2006243776A1 (en) 2006-11-09
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BRPI0611379A2 (pt) 2010-08-31
CN101212988A (zh) 2008-07-02
CN104306986A (zh) 2015-01-28
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US11957765B2 (en) 2024-04-16
PL2420256T3 (pl) 2016-12-30
WO2006119458A1 (en) 2006-11-09
JP6338560B2 (ja) 2018-06-06

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