JP4979067B2 - 神経代謝性疾患についての遺伝子治療 - Google Patents

神経代謝性疾患についての遺伝子治療 Download PDF

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JP4979067B2
JP4979067B2 JP2006514239A JP2006514239A JP4979067B2 JP 4979067 B2 JP4979067 B2 JP 4979067B2 JP 2006514239 A JP2006514239 A JP 2006514239A JP 2006514239 A JP2006514239 A JP 2006514239A JP 4979067 B2 JP4979067 B2 JP 4979067B2
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JP2006525363A (ja
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マルコ エイ． パッシーニ
グレッグ スチュアート
ジェイムス ダッジ
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    • 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
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    • C12N15/86—Viral vectors
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    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
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    • 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

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JP2006514239A 2003-05-01 2004-04-30 神経代謝性疾患についての遺伝子治療 Expired - Fee Related JP4979067B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US46719503P 2003-05-01 2003-05-01
US60/467,195 2003-05-01
US47572603P 2003-06-04 2003-06-04
US60/475,726 2003-06-04
PCT/US2004/013685 WO2004098648A1 (en) 2003-05-01 2004-04-30 Gene therapy for neurometabolic disorders

Related Child Applications (1)

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JP2011168292A Division JP2011251992A (ja) 2003-05-01 2011-08-01 神経代謝性疾患についての遺伝子治療

Publications (2)

Publication Number Publication Date
JP2006525363A JP2006525363A (ja) 2006-11-09
JP4979067B2 true JP4979067B2 (ja) 2012-07-18

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Family Applications (6)

Application Number Title Priority Date Filing Date
JP2006514239A Expired - Fee Related JP4979067B2 (ja) 2003-05-01 2004-04-30 神経代謝性疾患についての遺伝子治療
JP2011168292A Pending JP2011251992A (ja) 2003-05-01 2011-08-01 神経代謝性疾患についての遺伝子治療
JP2013196398A Expired - Fee Related JP5841574B2 (ja) 2003-05-01 2013-09-24 神経代謝性疾患についての遺伝子治療
JP2015106021A Expired - Fee Related JP6170519B2 (ja) 2003-05-01 2015-05-26 神経代謝性疾患についての遺伝子治療
JP2016205651A Expired - Lifetime JP6312771B2 (ja) 2003-05-01 2016-10-20 神経代謝性疾患についての遺伝子治療
JP2018000518A Pending JP2018058901A (ja) 2003-05-01 2018-01-05 神経代謝性疾患についての遺伝子治療

Family Applications After (5)

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JP2011168292A Pending JP2011251992A (ja) 2003-05-01 2011-08-01 神経代謝性疾患についての遺伝子治療
JP2013196398A Expired - Fee Related JP5841574B2 (ja) 2003-05-01 2013-09-24 神経代謝性疾患についての遺伝子治療
JP2015106021A Expired - Fee Related JP6170519B2 (ja) 2003-05-01 2015-05-26 神経代謝性疾患についての遺伝子治療
JP2016205651A Expired - Lifetime JP6312771B2 (ja) 2003-05-01 2016-10-20 神経代謝性疾患についての遺伝子治療
JP2018000518A Pending JP2018058901A (ja) 2003-05-01 2018-01-05 神経代謝性疾患についての遺伝子治療

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EP (3) EP1620133B1 (https=)
JP (6) JP4979067B2 (https=)
CY (1) CY1117457T1 (https=)
DK (1) DK1620133T3 (https=)
ES (1) ES2562490T3 (https=)
HU (1) HUE026861T2 (https=)
IL (2) IL171635A (https=)
PL (1) PL1620133T3 (https=)
PT (1) PT1620133E (https=)
SI (1) SI1620133T1 (https=)
WO (1) WO2004098648A1 (https=)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2011251992A (ja) * 2003-05-01 2011-12-15 Genzyme Corp 神経代謝性疾患についての遺伝子治療
US10913956B2 (en) 2003-05-01 2021-02-09 Genzyme Corporation Gene therapy for neurometabolic disorders

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US7829694B2 (en) 2002-11-26 2010-11-09 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
US7605249B2 (en) 2002-11-26 2009-10-20 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
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
US7994149B2 (en) 2003-02-03 2011-08-09 Medtronic, Inc. Method for treatment of Huntington's disease through intracranial delivery of sirna
US7732591B2 (en) 2003-11-25 2010-06-08 Medtronic, Inc. Compositions, devices and methods for treatment of huntington's disease through intracranial delivery of sirna
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
US7902352B2 (en) 2005-05-06 2011-03-08 Medtronic, Inc. Isolated nucleic acid duplex for reducing huntington gene expression
WO2006121960A2 (en) 2005-05-06 2006-11-16 Medtronic, Inc. Methods and sequences to suppress primate huntington gene expression
US9133517B2 (en) 2005-06-28 2015-09-15 Medtronics, Inc. Methods and sequences to preferentially suppress expression of mutated huntingtin
CN106729752A (zh) * 2006-02-08 2017-05-31 建新公司 A型尼曼‑皮克病的基因治疗
US9273356B2 (en) 2006-05-24 2016-03-01 Medtronic, Inc. Methods and kits for linking polymorphic sequences to expanded repeat mutations
US8324367B2 (en) 2006-11-03 2012-12-04 Medtronic, Inc. Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity
US9375440B2 (en) 2006-11-03 2016-06-28 Medtronic, Inc. Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity
US7988668B2 (en) 2006-11-21 2011-08-02 Medtronic, Inc. Microsyringe for pre-packaged delivery of pharmaceuticals
US7819842B2 (en) 2006-11-21 2010-10-26 Medtronic, Inc. Chronically implantable guide tube for repeated intermittent delivery of materials or fluids to targeted tissue sites
US11219696B2 (en) 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
HRP20250897T1 (hr) 2009-05-02 2025-09-26 Genzyme Corporation Genska terapija za neurodegenerativne poremećaje
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
AU2013296425B2 (en) 2012-08-01 2018-06-07 Nationwide Children's Hospital Intrathecal delivery of recombinant adeno-associated virus 9
EP4344741A3 (en) 2014-11-21 2024-08-28 The University of North Carolina at Chapel Hill Aav vectors targeted to the central nervous system
WO2016100575A1 (en) 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
EP3285788B1 (en) 2015-04-23 2024-12-18 University of Massachusetts Modulation of aav vector transgene expression
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
CN109415730A (zh) 2016-06-13 2019-03-01 北卡罗来纳大学教堂山分校 优化的cln1基因和表达盒以及它们的应用
DK3635009T3 (da) 2017-06-07 2026-03-30 Regeneron Pharma Sammensætninger og fremgangsmåder til internalisering af enzymer
AU2018346104B2 (en) 2017-10-03 2023-07-27 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
BR112020006661A2 (pt) 2017-10-03 2020-10-13 Prevail Therapeutics, Inc. terapias de genes para distúrbios lipossomais
CN112501208A (zh) 2017-10-03 2021-03-16 普利维尔治疗公司 用于溶酶体障碍的基因疗法
US12258597B2 (en) 2018-02-07 2025-03-25 Regeneron Pharmaceuticals, Inc. Methods and compositions for therapeutic protein delivery
CN112203697A (zh) 2018-04-13 2021-01-08 马萨诸塞大学 编码氨基己糖苷酶alpha和beta亚基的双顺反子AAV载体及其用途
BR112020021962A2 (pt) * 2018-04-30 2021-01-26 Amicus Therapeutics, Inc. construtos para terapia gênica e métodos de uso
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
US12448629B2 (en) 2018-10-05 2025-10-21 University Of Massachusetts rAAV vectors for the treatment of GM1 and GM2 gangliosidosis
CN113631182B (zh) 2018-10-10 2025-03-21 阿米库斯治疗学公司 二硫键稳定的多肽组合物和使用方法
KR20220022107A (ko) 2018-12-05 2022-02-24 아베오나 테라퓨틱스 인코퍼레이티드 유전자 전달을 위한 재조합 아데노-연관 바이러스 벡터
JP7664848B2 (ja) 2019-04-10 2025-04-18 プリベイル セラピューティクス，インコーポレーテッド リソソーム障害のための遺伝子療法
EP4284442A4 (en) * 2021-02-01 2025-04-23 The Trustees of The University of Pennsylvania COMPOSITIONS AND METHODS FOR TREATING NIEMANN PICK DISEASE TYPE A

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Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2011251992A (ja) * 2003-05-01 2011-12-15 Genzyme Corp 神経代謝性疾患についての遺伝子治療
JP2015178519A (ja) * 2003-05-01 2015-10-08 ジェンザイム・コーポレーション 神経代謝性疾患についての遺伝子治療
US10913956B2 (en) 2003-05-01 2021-02-09 Genzyme Corporation Gene therapy for neurometabolic disorders

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Publication number Publication date
PT1620133E (pt) 2016-03-31
JP2017008118A (ja) 2017-01-12
JP6170519B2 (ja) 2017-07-26
EP2269658A1 (en) 2011-01-05
WO2004098648A1 (en) 2004-11-18
IL252829A0 (en) 2017-08-31
EP3513814A1 (en) 2019-07-24
EP1620133A1 (en) 2006-02-01
HUE026861T2 (en) 2016-07-28
PL1620133T3 (pl) 2016-05-31
IL171635A (en) 2017-06-29
EP2269658B1 (en) 2018-12-19
JP5841574B2 (ja) 2016-01-13
JP2015178519A (ja) 2015-10-08
JP2018058901A (ja) 2018-04-12
JP2014040433A (ja) 2014-03-06
JP2011251992A (ja) 2011-12-15
ES2562490T3 (es) 2016-03-04
CY1117457T1 (el) 2017-04-26
EP1620133B1 (en) 2015-12-09
JP2006525363A (ja) 2006-11-09
JP6312771B2 (ja) 2018-04-18
SI1620133T1 (sl) 2016-03-31
DK1620133T3 (en) 2016-02-29

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