CN112501208A - 用于溶酶体障碍的基因疗法 - Google Patents

用于溶酶体障碍的基因疗法 Download PDF

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CN112501208A
CN112501208A CN202011187145.9A CN202011187145A CN112501208A CN 112501208 A CN112501208 A CN 112501208A CN 202011187145 A CN202011187145 A CN 202011187145A CN 112501208 A CN112501208 A CN 112501208A
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nucleic acid
vector
sequence
itr
raav
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阿萨·阿贝利奥维奇
劳拉·赫克曼
赫维·莱茵
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Privelle Therapeutics
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CN202011187145.9A 2017-10-03 2018-10-03 用于溶酶体障碍的基因疗法 Pending CN112501208A (zh)

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CN113846122A (zh) * 2021-08-16 2021-12-28 中国医学科学院医学生物学研究所 一种过表达snca的腺相关病毒载体aav-snca、制备方法及其应用

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Publication number Priority date Publication date Assignee Title
CN111465691A (zh) 2017-10-03 2020-07-28 普利维尔治疗公司 用于溶酶体障碍的基因疗法
JP7254815B2 (ja) 2017-10-03 2023-04-10 プリベイル セラピューティクス,インコーポレーテッド ライソゾーム病の遺伝子治療
KR102709597B1 (ko) 2017-10-03 2024-09-26 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
DK3850089T5 (da) 2019-02-04 2024-08-19 Freeline Therapeutics Ltd Polynukleotider
KR20210131370A (ko) 2019-02-22 2021-11-02 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Grn-연관 성인-발병 신경퇴화의 치료를 위한 재조합 아데노-연관 바이러스
AU2020273182B2 (en) 2019-04-10 2025-11-20 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
CN114026115A (zh) 2019-04-10 2022-02-08 普利维尔治疗公司 用于溶酶体病症的基因疗法
EP4013870A1 (en) * 2019-08-13 2022-06-22 Alnylam Pharmaceuticals, Inc. Small ribosomal protein subunit 25 (rps25) irna agent compositions and methods of use thereof
JP2023537904A (ja) 2020-08-10 2023-09-06 プリベイル セラピューティクス,インコーポレーテッド 神経変性障害のための遺伝子療法
WO2022035903A2 (en) 2020-08-10 2022-02-17 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
WO2022082017A2 (en) 2020-10-15 2022-04-21 Prevail Therapeutics, Inc. Recombinant adeno-associated virus compositions and methods for producing same
US20240044869A1 (en) 2020-10-15 2024-02-08 Prevail Therapeutics, Inc. Assay for measuring potency of gene therapy drug product
WO2022097727A1 (ja) * 2020-11-06 2022-05-12 住友ファーマ株式会社 Rps25遺伝子の発現及び/又は機能調節剤
US20250382611A1 (en) * 2021-06-21 2025-12-18 Uniqure Biopharma B.V. Gene constructs for silencing alpha-synuclein and uses thereof
US20240269161A1 (en) * 2021-06-28 2024-08-15 The Board Of Regents Of The University Of Texas System Methods and compositions for tau reduction gene therapy
WO2023178338A2 (en) * 2022-03-18 2023-09-21 University Of Florida Research Foundation, Incorporated Methods and compositions for treating tmem43 related cardiomyopathy with a viral vector
TW202515584A (zh) * 2023-10-11 2025-04-16 大陸商上海舶望製藥有限公司 用於抑制微管相關蛋白tau(mapt)表達的組合物和方法
WO2025160429A1 (en) * 2024-01-26 2025-07-31 Genzyme Corporation Artificial micrornas targeting snca

Citations (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001083692A2 (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
US20010051611A1 (en) * 1996-09-06 2001-12-13 Chiron Corporation. Methods and compositions for liver specific delivery of therapeutic molecules using recombinant AAV vectors
US20030100115A1 (en) * 2000-04-20 2003-05-29 Kenneth Raj Cytotoxic agents
US20080249058A1 (en) * 2007-04-05 2008-10-09 Erik Roberson Agents that reduce neuronal overexcitation
CN101448944A (zh) * 2006-03-17 2009-06-03 西伦蒂斯公司 Cns病症的治疗
CN102181480A (zh) * 2001-11-13 2011-09-14 宾夕法尼亚州立大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
WO2012027713A2 (en) * 2010-08-26 2012-03-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of snca
CN103189507A (zh) * 2010-10-27 2013-07-03 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
CN103562396A (zh) * 2011-02-22 2014-02-05 Isis创新公司 用于无脉络膜的基因疗法的aav载体
US20140051748A1 (en) * 2010-08-25 2014-02-20 Asa Abeliovich OPTIMIZED miRNA CONSTRUCTS
CN105431153A (zh) * 2013-07-11 2016-03-23 纽约市哥伦比亚大学理事会 使tau表达沉默的微RNA
CN105431170A (zh) * 2013-04-08 2016-03-23 爱荷华大学研究基金会 嵌合腺相关病毒/博卡病毒细小病毒载体
WO2016151523A1 (en) * 2015-03-25 2016-09-29 Università Degli Studi Di Trento Rna interference mediated therapy for neurodegenerative diseases
WO2016179497A1 (en) * 2015-05-07 2016-11-10 Shire Human Genetic Therapies, Inc. Glucocerebrosidase gene therapy for parkinson's disease
CN106456711A (zh) * 2014-02-25 2017-02-22 曼彻斯特大学 使用基因疗法治疗视网膜退化
CN107106689A (zh) * 2014-11-05 2017-08-29 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸

Family Cites Families (58)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5879680A (en) 1987-12-23 1999-03-09 The United States Of America As Represented By The Department Of Health And Human Services Cloned DNA for synthesizing unique glucocerebrosidase
EP1112090A2 (en) 1998-09-09 2001-07-04 Myelos Corporation Method of stimulating prosaposin receptor activity
AU6234099A (en) 1998-10-16 2000-05-08 Introgene B.V. Gene therapy of alzheimer's disease by delivery of an encoded apoliprotein
DE69941905D1 (de) 1998-11-10 2010-02-25 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
US6696272B1 (en) 1999-06-02 2004-02-24 Hsc Research & Development Limited Partnership Products and methods for gaucher disease therapy
AU2001253233B2 (en) 2000-04-06 2006-12-07 Kos Pharmaceuticals, Inc. Compounds and methods for lowering cholesterol levels without inducing hypertriglyceridemia
AU2001290984A1 (en) 2000-09-18 2002-04-02 Genzyme Corporation Expression vectors containing hybrid ubiquitin promoters
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
US20030133924A1 (en) 2001-12-21 2003-07-17 Novazyme Pharmaceuticals, Inc. Highly phosphorylated acid beta-glucocerebrosidase and methods of treating gaucher's disease
US20060292117A1 (en) 2002-04-17 2006-12-28 Loiler Scott A Improved rAAv vectors
PL1620133T3 (pl) 2003-05-01 2016-05-31 Genzyme Corp Terapia genowa dla zaburzeń neurometabolicznych
WO2006036465A2 (en) 2004-09-03 2006-04-06 University Of Florida Compositions and methods for treating cystic fibrosis
EP1799826B1 (en) 2004-09-29 2009-08-12 Children's Memorial Hospital siRNA-MEDIATED GENE SILENCING OF ALPHA SYNUCLEIN
MX2007015602A (es) 2005-06-08 2008-02-21 Amicus Therapeutics Inc Tratamiento de trastornos del sistema nervioso central asociados con mutaciones en genes que codifican las enzimas lisosomicas.
EP2687223B2 (en) 2006-05-30 2024-11-20 Mayo Foundation For Medical Education And Research Detecting and treating dementia
JP5123936B2 (ja) 2006-05-30 2013-01-23 メイヨ・ファウンデーション・フォー・メディカル・エデュケーション・アンド・リサーチ 認知症の検出および治療
PL2489733T3 (pl) 2006-06-07 2019-08-30 Genzyme Corporation Terapia genowa w stwardnieniu zanikowym bocznym i innych zaburzeniach rdzenia kręgowego
EP3456733A1 (en) 2007-05-11 2019-03-20 Genzyme Corporation Methods of producing a secreted protein
HUE027278T2 (en) 2007-05-16 2016-11-28 Brigham & Womens Hospital Inc Treatment of synucleinopathies
US20090176729A1 (en) * 2007-12-14 2009-07-09 Alnylam Pharmaceuticals, Inc. Method of treating neurodegenerative disease
CN102006882B (zh) 2008-01-16 2017-06-06 神经动力公司 利用颗粒体蛋白前体(pgrn)治疗神经变性疾病
WO2009120978A2 (en) 2008-03-27 2009-10-01 The Ohio State University Treatment of metabolic-related disorders using hypothalamic gene transfer of bdnf and compositions therfor
DK3421603T3 (da) 2009-05-02 2022-01-10 Genzyme Corp Genterapi for neurodegenerative forstyrrelser
WO2010129791A1 (en) * 2009-05-06 2010-11-11 University Of Medicine And Dentistry Of New Jersey Rna targeting in alpha-synucleinopathies
US9102949B2 (en) * 2010-04-23 2015-08-11 University Of Massachusetts CNS targeting AAV vectors and methods of use thereof
EP2640407A4 (en) 2010-11-16 2014-07-09 Denis G Kay PROCESS FOR INCREASING THE EXPRESSION AND ACTIVITY OF NEPRILYSIN
ES2773480T3 (es) 2011-05-09 2020-07-13 Eip Pharma Llc Composiciones y métodos para tratar la enfermedad de Alzheimer
HK1206601A1 (en) 2012-04-02 2016-01-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and proteins associated with human disease
RU2673484C2 (ru) 2012-05-18 2018-11-27 Юниверсити Оф Айова Рисерч Фаундейшн Способы и композиции для лечения амилоидных отложений
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
BR112015009746A2 (pt) 2012-11-05 2017-08-15 Genzyme Corp Agente que aumenta atividade de glicocerebrosidase em mamíferos
HK1222093A1 (zh) 2013-05-15 2017-06-23 明尼苏达大学董事会 腺相关病毒介导的基因向中枢神经系统转移
US9347107B2 (en) 2013-05-23 2016-05-24 Norman Z Lai Vector containing multiple nucleotide sequences for the expression of enzymes
US9574184B2 (en) 2013-09-25 2017-02-21 Children's Hospital Medical Center Lysosomal protein targeting sequence and therapeutic applications of same
CA2927366A1 (en) 2013-10-24 2015-04-30 Uniqure Ip B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
US10603364B2 (en) 2014-08-11 2020-03-31 Shire Human Genetic Therapies, Inc. Lysosomal targeting and uses thereof
EP3221456B1 (en) 2014-11-21 2021-09-22 University of Florida Research Foundation, Inc. Genome-modified recombinant adeno-associated virus vectors
US20170035860A1 (en) 2015-04-02 2017-02-09 Alexander C. Flynn Compositions and methods for treatment of neurogenerative diseases
US11020443B2 (en) 2015-04-23 2021-06-01 University Of Massachusetts Modulation of AAV vector transgene expression
EP3091087A1 (en) 2015-05-08 2016-11-09 Fundació Institut d'Investigació en Ciències de la Salut Germans Trias i Pujol Method for in vitro diagnosis of synucleinopathies using alpha-synuclein gene transcripts
MX2017014443A (es) 2015-05-15 2018-08-01 Univ Minnesota Adeno-asociado para administracion terapeutica al sistema nervioso central.
WO2017075338A2 (en) 2015-10-29 2017-05-04 Voyager Therapeutics, Inc. Delivery of central nervous system targeting polynucleotides
BR112018007453A2 (pt) 2015-11-05 2018-10-23 Bamboo Therapeutics Inc genes modificados de ataxia de friedreich e vetores para a terapia gênica
CA3011939A1 (en) 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
FI3411484T3 (fi) 2016-02-05 2023-11-15 Univ Emory Yksisäikeisen tai itsekomplementaarisen adenoassosioidun viruksen 9 injektio serebrospinaaliseen fluidiin
US11066456B2 (en) 2016-02-25 2021-07-20 Washington University Compositions comprising TREM2 and methods of use thereof
WO2017151884A1 (en) 2016-03-02 2017-09-08 The Children's Hospital Of Philadelphia Therapy for frontotemporal dementia
KR102850887B1 (ko) 2017-08-03 2025-08-28 보이저 테라퓨틱스, 인크. Aav의 전달을 위한 조성물 및 방법
JP7254815B2 (ja) 2017-10-03 2023-04-10 プリベイル セラピューティクス,インコーポレーテッド ライソゾーム病の遺伝子治療
CN111465691A (zh) 2017-10-03 2020-07-28 普利维尔治疗公司 用于溶酶体障碍的基因疗法
KR102709597B1 (ko) 2017-10-03 2024-09-26 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
US20200283800A1 (en) 2017-10-23 2020-09-10 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative diseases
CN111819281B (zh) 2017-10-23 2024-12-31 普利维尔治疗公司 用于神经变性疾病的基因疗法
CA3121211A1 (en) 2018-11-28 2020-06-04 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disease
AU2020273182B2 (en) 2019-04-10 2025-11-20 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
CN114026115A (zh) 2019-04-10 2022-02-08 普利维尔治疗公司 用于溶酶体病症的基因疗法
WO2020210615A1 (en) 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
US20220211871A1 (en) 2019-04-10 2022-07-07 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders

Patent Citations (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20010051611A1 (en) * 1996-09-06 2001-12-13 Chiron Corporation. Methods and compositions for liver specific delivery of therapeutic molecules using recombinant AAV vectors
US20030100115A1 (en) * 2000-04-20 2003-05-29 Kenneth Raj Cytotoxic agents
WO2001083692A2 (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
CN102181480A (zh) * 2001-11-13 2011-09-14 宾夕法尼亚州立大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
CN101448944A (zh) * 2006-03-17 2009-06-03 西伦蒂斯公司 Cns病症的治疗
US20080249058A1 (en) * 2007-04-05 2008-10-09 Erik Roberson Agents that reduce neuronal overexcitation
US20140051748A1 (en) * 2010-08-25 2014-02-20 Asa Abeliovich OPTIMIZED miRNA CONSTRUCTS
WO2012027713A2 (en) * 2010-08-26 2012-03-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of snca
CN103189507A (zh) * 2010-10-27 2013-07-03 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
CN103562396A (zh) * 2011-02-22 2014-02-05 Isis创新公司 用于无脉络膜的基因疗法的aav载体
CN105431170A (zh) * 2013-04-08 2016-03-23 爱荷华大学研究基金会 嵌合腺相关病毒/博卡病毒细小病毒载体
CN105431153A (zh) * 2013-07-11 2016-03-23 纽约市哥伦比亚大学理事会 使tau表达沉默的微RNA
CN106456711A (zh) * 2014-02-25 2017-02-22 曼彻斯特大学 使用基因疗法治疗视网膜退化
CN107106689A (zh) * 2014-11-05 2017-08-29 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
WO2016151523A1 (en) * 2015-03-25 2016-09-29 Università Degli Studi Di Trento Rna interference mediated therapy for neurodegenerative diseases
WO2016179497A1 (en) * 2015-05-07 2016-11-10 Shire Human Genetic Therapies, Inc. Glucocerebrosidase gene therapy for parkinson's disease

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
SIMAN R等: "A rapid gene delivery-based mouse model for early-stage Alzheimer disease-type tauopathy", 《J NEUROPATHOL EXP NEUROL》, vol. 72, no. 11, pages 1062 - 1071 *
VINCENT M等: "Comparison of the efficacy of five adeno-associated virus vectors for transducing dorsal raphé nucleus cells in the mouse", 《 J NEUROSCI METHODS》, pages 189 - 192 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113846122A (zh) * 2021-08-16 2021-12-28 中国医学科学院医学生物学研究所 一种过表达snca的腺相关病毒载体aav-snca、制备方法及其应用
CN113846122B (zh) * 2021-08-16 2023-08-25 中国医学科学院医学生物学研究所 一种过表达snca的腺相关病毒载体aav-snca、制备方法及其应用

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