CN112553210B - 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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CN112553210B
CN112553210B CN202011191794.6A CN202011191794A CN112553210B CN 112553210 B CN112553210 B CN 112553210B CN 202011191794 A CN202011191794 A CN 202011191794A CN 112553210 B CN112553210 B CN 112553210B
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nucleic acid
seq
raav
vector
itr
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CN112553210A (zh
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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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Privelle Therapeutics
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CN202011191794.6A 2017-10-03 2018-10-03 用于溶酶体障碍的基因疗法 Active CN112553210B (zh)

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CN202011191794.6A CN112553210B (zh) 2017-10-03 2018-10-03 用于溶酶体障碍的基因疗法

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US201762567310P 2017-10-03 2017-10-03
US201762567296P 2017-10-03 2017-10-03
US201762567311P 2017-10-03 2017-10-03
US201762567301P 2017-10-03 2017-10-03
US201762567319P 2017-10-03 2017-10-03
US62/567,311 2017-10-03
US62/567,319 2017-10-03
US62/567,310 2017-10-03
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Families Citing this family (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JOP20190248A1 (ar) 2017-04-21 2019-10-20 Amgen Inc بروتينات ربط مولد ضد trem2 واستخداماته
TWI868807B (zh) 2017-08-03 2025-01-01 美商阿列克特有限責任公司 抗trem2抗體及其使用方法
CN112553210B (zh) 2017-10-03 2024-09-24 普利维尔治疗公司 用于溶酶体障碍的基因疗法
BR112020006633A2 (pt) 2017-10-03 2020-10-06 Prevail Therapeutics, Inc. terapias genéticas para distúrbios lisossômicos
CA3078501A1 (en) 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
PL3850089T3 (pl) 2019-02-04 2024-05-06 Freeline Therapeutics Limited Polinukleotydy
MX2021010134A (es) 2019-02-22 2021-09-23 Univ Pennsylvania Virus adenoasociado recombinante para el tratamiento de la neurodegeneracion de inicio en adultos asociada a grn.
SG11202108480UA (en) * 2019-02-26 2021-09-29 Univ Pennsylvania Compositions useful in treatment of krabbe disease
KR20220006527A (ko) 2019-04-10 2022-01-17 프리베일 테라퓨틱스, 인크. 리소좀 장애에 대한 유전자 요법
US11999974B2 (en) * 2019-04-10 2024-06-04 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
GB201913974D0 (en) * 2019-09-27 2019-11-13 King S College London Vector
MX2022004812A (es) * 2019-10-22 2023-02-23 Applied Genetic Tech Corporation Sistemas de virus adeno-asociados (aav) para el tratamiento de enfermedades o trastornos neurodegenerativos asociados a la progranulina.
US20230021959A1 (en) * 2019-12-05 2023-01-26 The Trustees Of Columbia University In The City Of New York Stabilization of Retromer for the Treatment of Alzheimer's Disease and Other Neurodegenerative Disorders
WO2021163681A2 (en) * 2020-02-14 2021-08-19 The Trustees Of Columbia University In The City Of New York Combination of retromer pharmacological chaperones and exogenous retromer for the treatment of alzheimer's disease and other neurodegenerative diseases and disorders
US20230210941A1 (en) * 2020-05-12 2023-07-06 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of krabbe disease
ES3057565T3 (en) * 2020-07-23 2026-03-03 Univ North Carolina Chapel Hill Optimized slc13a5 genes and expression cassettes and their use
BR112023001456A2 (pt) 2020-07-27 2023-04-11 Voyager Therapeutics Inc Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase
IL299811A (en) 2020-07-29 2023-03-01 Freeline Therapeutics Ltd polypeptide
CN116113701A (zh) * 2020-08-03 2023-05-12 普利维尔治疗公司 编码parkin的aav载体及其用途
MX2023001562A (es) 2020-08-10 2023-05-03 Prevail Therapeutics Inc Terapias genicas para trastornos lisosomales.
CN116437968A (zh) * 2020-08-10 2023-07-14 普利维尔治疗公司 用于神经退行性病症的基因疗法
JP2023545462A (ja) 2020-10-14 2023-10-30 デナリ セラピューティクス インコーポレイテッド 前頭側頭型認知症を治療及びモニタリングするための方法
IL301858A (en) 2020-10-15 2023-06-01 Prevail Therapeutics Inc Assay for measuring potency of gene therapy drug product
JP2023545830A (ja) * 2020-10-15 2023-10-31 プリベイル セラピューティクス，インコーポレーテッド 組換えアデノ随伴ウイルス組成物及びその生産方法
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
WO2022271944A2 (en) * 2021-06-23 2022-12-29 Avrobio, Inc. Compositions and methods for treatment of gaucher disease
US20250049955A1 (en) 2021-11-17 2025-02-13 Voyager Therapeutics, Inc. Compositons and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
JP1727698S (ja) * 2021-12-20 2022-10-19 ゴルフボールケース
JP2025516136A (ja) 2022-04-19 2025-05-27 シャンハイ ヴァイタルゲン バイオファーマ カンパニー リミテッド 神経変性疾患の治療のための組み換えａａｖベクター
EP4598940A1 (en) 2022-10-04 2025-08-13 Eli Lilly and Company Gene therapy for trem2-associated diseases and disorders
JP2025534899A (ja) * 2022-10-08 2025-10-21 リンイ バイオテック カンパニー リミテッド ＧＣａｓｅ欠乏関連疾患を治療するためのポリヌクレオチド
GB202216168D0 (en) 2022-10-31 2022-12-14 UCB Biopharma SRL Route of administration
AU2023427408A1 (en) * 2023-02-02 2025-09-04 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
JP2026509301A (ja) 2023-03-14 2026-03-17 ユーシービー バイオファルマ エスアールエル 遺伝子治療
WO2025039622A1 (zh) * 2023-08-23 2025-02-27 上海金珂博生物技术有限公司 经修饰的腺相关病毒载体及其在治疗中枢神经系统疾病中的用途
CN118086341B (zh) * 2024-04-25 2024-08-02 上海凌医生物科技有限公司 在肝脏中高表达人源葡萄糖脑苷脂酶基因的表达框

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016081927A2 (en) * 2014-11-21 2016-05-26 University Of Florida Research Foundation, Inc. Genome-modified recombinant adeno-associated virus vectors
WO2017151884A1 (en) * 2016-03-02 2017-09-08 The Children's Hospital Of Philadelphia Therapy for frontotemporal dementia

Family Cites Families (87)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS6182035A (ja) * 1984-09-27 1986-04-25 Toshiba Corp 制振装置
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
US5490559A (en) * 1994-07-20 1996-02-13 Dinulescu; Horia A. Heat exchanger with finned partition walls
US20020068285A1 (en) * 1996-01-11 2002-06-06 Frudakis Tony N. Compositions and methods for the therapy and diagnosis of breast cancer
US6521225B1 (en) 1996-09-06 2003-02-18 Chiron Corporation AAV vectors
US6538119B2 (en) 1997-11-18 2003-03-25 Abbott Laboratories Reagents and methods useful for detecting diseases of the breast
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
ATE454445T1 (de) 1998-11-10 2010-01-15 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
DE19937800B4 (de) * 1999-08-10 2005-06-16 Gea Energietechnik Gmbh Anlage zur Kondensation von Dampf
JP2003530835A (ja) 2000-04-06 2003-10-21 ケイオウエス ファーマシューティカルズ インコーポレイテッド 高トリグリセリド血症を誘発することなくコレステロールレベルを低下するための化合物と方法
GB0009887D0 (en) 2000-04-20 2000-06-07 Btg Int Ltd Cytotoxic agents
AU2001255575B2 (en) 2000-04-28 2006-08-31 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
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
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
US6691742B1 (en) * 2003-04-25 2004-02-17 Thomas L. Cooper Method and apparatus for supporting a pipe
ES2562490T3 (es) 2003-05-01 2016-03-04 Genzyme Corporation Terapia génica para trastornos neurometabólicos
WO2005049676A1 (de) 2003-11-21 2005-06-02 Basf Aktiengesellschaft Polyalkylenoxidgruppen und quartäre stickstoffatome enthaltende copolymere
CN101443046A (zh) * 2004-06-23 2009-05-27 建新公司 治疗多囊病的方法和组合物
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.
GB0605337D0 (en) 2006-03-17 2006-04-26 Genomica Sau Treatment of CNS conditions
EP2687223B2 (en) 2006-05-30 2024-11-20 Mayo Foundation For Medical Education And Research Detecting and treating dementia
WO2008019187A2 (en) * 2006-05-30 2008-02-14 Mayo Foundation For Medical Education And Research Detecting and treating dementia
CN101460621B (zh) * 2006-06-07 2016-01-27 建新公司 肌萎缩性侧索硬化和其他脊髓失调症的基因治疗
DE102006029773B3 (de) * 2006-06-27 2007-07-12 Gea Energietechnik Gmbh Verfahren zur Errichtung einer Kondensationsanlage
CN101583621A (zh) * 2006-07-20 2009-11-18 诺华有限公司 治疗、诊断或检测癌症的amigo-2抑制剂
US20080249058A1 (en) 2007-04-05 2008-10-09 Erik Roberson Agents that reduce neuronal overexcitation
US8962273B2 (en) 2007-05-11 2015-02-24 Genzyme Corporation Methods of producing a secreted protein
US8454954B2 (en) 2007-05-16 2013-06-04 The Brigham And Women's Hospital, Inc. Treatment of synucleinopathies
EP3127549B1 (en) * 2007-06-22 2019-12-18 Children's Medical Center Corporation Methods and uses thereof of a fragment of saposin a
WO2009079399A2 (en) 2007-12-14 2009-06-25 Alnylam Pharmaceuticals, Inc. Method of treating neurodegenerative disease
EP2249861B1 (en) 2008-01-16 2016-07-06 Neurodyn Life Sciences Inc. Progranulin for use in treating parkinson's disease or alzheimer's disease
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
JP5879256B2 (ja) 2009-05-02 2016-03-08 ジェンザイム・コーポレーション 神経変性障害のための遺伝子治療
US9255266B2 (en) 2009-05-06 2016-02-09 Rutgers, The State University Of New Jersey RNA targeting in alpha-synucleinopathies
CN102858985A (zh) * 2009-07-24 2013-01-02 西格马-奥尔德里奇有限责任公司 基因组编辑方法
EP2513137B1 (en) * 2009-12-17 2018-02-28 Children's Medical Center Corporation Saposin-a derived peptides and uses thereof
EP3567106A1 (en) 2010-04-23 2019-11-13 University of Massachusetts Cns targeting aav vectors and methods of use thereof
US9290759B2 (en) 2010-08-25 2016-03-22 The Trustees Of Columbia University In The City Of New York Optimized miRNA constructs
WO2012027713A2 (en) 2010-08-26 2012-03-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of snca
CN107828820B (zh) 2010-10-27 2022-06-07 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
WO2012065248A1 (en) 2010-11-16 2012-05-24 Kay Denis G Method for increasing neprilysin expression and activity
CN103842362B (zh) 2011-05-09 2017-05-24 爱普制药有限责任公司 用于治疗阿尔茨海默氏病的组合物和方法
WO2013151663A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of membrane proteins
EP2850195B1 (en) 2012-05-18 2020-01-22 University of Iowa Research Foundation Methods and compositions for treating amyloid deposits
CA2875618C (en) 2012-07-11 2021-04-27 Sangamo Biosciences, Inc. Methods and compositions for the treatment of lysosomal storage diseases
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
US9828587B2 (en) 2013-04-08 2017-11-28 University Of Iowa Research Foundation Chimeric adeno-associated virus/ bocavirus parvovirus vector
KR20160010526A (ko) 2013-05-15 2016-01-27 리젠츠 오브 더 유니버시티 오브 미네소타 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달
US9347107B2 (en) 2013-05-23 2016-05-24 Norman Z Lai Vector containing multiple nucleotide sequences for the expression of enzymes
US9951330B2 (en) 2013-07-11 2018-04-24 The Trustees Of Columbia University In The City Of New York Micrornas that silence tau expression
US9574184B2 (en) 2013-09-25 2017-02-21 Children's Hospital Medical Center Lysosomal protein targeting sequence and therapeutic applications of same
SG10201913611QA (en) * 2014-08-08 2020-03-30 Alector Llc Anti-trem2 antibodies and methods of use thereof
WO2016025523A1 (en) * 2014-08-11 2016-02-18 Shire Human Genetic Therapies, Inc. Lysosomal targeting and uses thereof
WO2016036403A1 (en) * 2014-09-05 2016-03-10 Population Diagnostics Inc. Methods and compositions for inhibiting and treating neurological conditions
ES2714130T3 (es) * 2015-02-02 2019-05-27 Procter & Gamble Composición detergente
US10513701B2 (en) 2015-03-25 2019-12-24 Università Degli Studi Di Trento RNA interference mediated therapy for neurodegenerative diseases
US20170035860A1 (en) 2015-04-02 2017-02-09 Alexander C. Flynn Compositions and methods for treatment of neurogenerative diseases
EP4491733A3 (en) * 2015-04-23 2025-03-26 University of Massachusetts Modulation of aav vector transgene expression
WO2016179497A1 (en) 2015-05-07 2016-11-10 Shire Human Genetic Therapies, Inc. Glucocerebrosidase gene therapy for parkinson's disease
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
CA2986252A1 (en) 2015-05-15 2016-11-24 Regents Of The University Of Minnesota Adeno-associated virus vector for delivery of iduronate-2-sulfatase to the central nervous system
EP3356507A1 (en) * 2015-10-02 2018-08-08 The Procter and Gamble Company Cleaning pad, cleaning implement, and a method of improving shine of a hard surface using the cleaning pad or cleaning implement with a cleaning composition
WO2017075338A2 (en) 2015-10-29 2017-05-04 Voyager Therapeutics, Inc. Delivery of central nervous system targeting polynucleotides
PE20181156A1 (es) 2015-11-05 2018-07-19 Bamboo Therapeutics Inc Genes de ataxia de friedreich modificados y vectores para terapia genica
WO2017136536A1 (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
US12070510B2 (en) 2016-02-05 2024-08-27 Emory University Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid
US11066456B2 (en) 2016-02-25 2021-07-20 Washington University Compositions comprising TREM2 and methods of use thereof
ES2802454T3 (es) * 2016-04-08 2021-01-19 Procter & Gamble Composición de limpieza para lavavajillas
TWI832036B (zh) 2017-08-03 2024-02-11 美商航海家醫療公司 用於aav之遞送之組合物及方法
BR112020006633A2 (pt) 2017-10-03 2020-10-06 Prevail Therapeutics, Inc. terapias genéticas para distúrbios lisossômicos
CA3078501A1 (en) 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
CN112553210B (zh) 2017-10-03 2024-09-24 普利维尔治疗公司 用于溶酶体障碍的基因疗法
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
BR112021010234A2 (pt) 2018-11-28 2021-08-24 Prevail Therapeutics, Inc. Terapias de gene para doença neurodegenerativa
US11999974B2 (en) 2019-04-10 2024-06-04 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
US20220211871A1 (en) 2019-04-10 2022-07-07 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
KR20220006527A (ko) 2019-04-10 2022-01-17 프리베일 테라퓨틱스, 인크. 리소좀 장애에 대한 유전자 요법
CA3134841A1 (en) 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
CN116437968A (zh) 2020-08-10 2023-07-14 普利维尔治疗公司 用于神经退行性病症的基因疗法

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016081927A2 (en) * 2014-11-21 2016-05-26 University Of Florida Research Foundation, Inc. Genome-modified recombinant adeno-associated virus vectors
WO2017151884A1 (en) * 2016-03-02 2017-09-08 The Children's Hospital Of Philadelphia Therapy for frontotemporal dementia

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Teng Jiang等.Upregulation of TREM2 Ameliorates Neuropathology and Rescues Spatial Cognitive Impairment in a Transgenic Mouse Model of Alzheimer’s Disease.《Neuropsychopharmacology》.2014,第39卷第2949–2962页. *
Upregulation of TREM2 Ameliorates Neuropathology and Rescues Spatial Cognitive Impairment in a Transgenic Mouse Model of Alzheimer’s Disease;Teng Jiang等;《Neuropsychopharmacology》;第39卷;第2949–2962页 *

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