JP2021522273A - Ｃｎｓ変性のための遺伝子治療法 - Google Patents

Ｃｎｓ変性のための遺伝子治療法 Download PDF

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JP2021522273A
JP2021522273A JP2020560275A JP2020560275A JP2021522273A JP 2021522273 A JP2021522273 A JP 2021522273A JP 2020560275 A JP2020560275 A JP 2020560275A JP 2020560275 A JP2020560275 A JP 2020560275A JP 2021522273 A JP2021522273 A JP 2021522273A
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gene
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gene therapy
transgene
protein
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JPWO2019210325A5 (https=
JP2021522273A5 (https=
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アンナヒータ ケラバラ
アンナヒータ ケラバラ
パベン バッティプロール
パベン バッティプロール
ラジ プラバカール
ラジ プラバカール
ロデリック ウォン
ロデリック ウォン
ピラチップ プラタムスワン
ピラチップ プラタムスワン
ナヴィーン ヤラマンチ
ナヴィーン ヤラマンチ
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ロケット ファーマシューティカルズ リミテッド
ロケット ファーマシューティカルズ リミテッド
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Publication of JP2021522273A publication Critical patent/JP2021522273A/ja
Publication of JPWO2019210325A5 publication Critical patent/JPWO2019210325A5/ja
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JP2020560275A 2018-04-27 2019-04-29 Ｃｎｓ変性のための遺伝子治療法 Pending JP2021522273A (ja)

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JP2024021996A JP2024059727A (ja) 2018-04-27 2024-02-16 Ｃｎｓ変性のための遺伝子治療法

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US201862664006P 2018-04-27 2018-04-27
US62/664,006 2018-04-27
PCT/US2019/029744 WO2019210325A1 (en) 2018-04-27 2019-04-29 Gene therapy for cns degeneration

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JP2021522273A true JP2021522273A (ja) 2021-08-30
JPWO2019210325A5 JPWO2019210325A5 (https=) 2022-05-02
JP2021522273A5 JP2021522273A5 (https=) 2022-05-02

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JP2024021996A Pending JP2024059727A (ja) 2018-04-27 2024-02-16 Ｃｎｓ変性のための遺伝子治療法

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US (1) US12378576B2 (https=)
EP (1) EP3810781A4 (https=)
JP (2) JP2021522273A (https=)
KR (1) KR20210005889A (https=)
CN (1) CN112368390B (https=)
AU (1) AU2019261646A1 (https=)
BR (1) BR112020021903A2 (https=)
CA (1) CA3096293A1 (https=)
IL (1) IL278213B2 (https=)
MX (1) MX2020011386A (https=)
RU (1) RU2020134965A (https=)
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Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3021572A1 (en) 2016-04-20 2017-10-26 Centro De Investigaciones Energeticas, Medioambientales Y Tecnologicas Compositions and methods for enhanced gene expression of pklr
EP3697452A4 (en) 2017-10-16 2021-11-24 Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, O.A., M.P. LENTIVIRAL VECTORS FOR ADMINISTRATION OF PKLR TO TREAT PYRUVATE KINASE DEFECTS
EP3720507A4 (en) * 2017-12-07 2021-08-25 Ramot at Tel-Aviv University Ltd. TREATMENT OF PARKINSON PATIENTS WITH MUTATIONS IN THE LRRK2 GENE
BR112020020841A2 (pt) 2018-04-11 2021-01-19 Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus Composições e métodos para transplante de célulastronco
CA3096293A1 (en) 2018-04-27 2019-10-31 Rocket Pharmaceuticals, Ltd. Gene therapy for cns degeneration
JP2023535121A (ja) * 2020-05-20 2023-08-16 スペースクラフト セブン リミテッド ライアビリティ カンパニー 操作されたパーキン及びその使用
JP2023526449A (ja) * 2020-05-21 2023-06-21 ニスノバイオ ジーティー，エルエルシー パーキンソン病を治療するための、増加した活性を有するパーキン変異体の遺伝子治療送達
CA3190720A1 (en) * 2020-08-03 2022-02-10 Prevail Therapeutics, Inc. Aav vectors encoding parkin and uses thereof
JP2023538519A (ja) 2020-08-07 2023-09-08 スペースクラフト セブン リミテッド ライアビリティ カンパニー Ａａｖベクターを使用したプラコフィリン－２（ｐｋｐ２）遺伝子治療
WO2022040530A2 (en) * 2020-08-21 2022-02-24 Capsida, Inc. Virus compositions for treating mucopolysaccharidosis ii
WO2022066911A1 (en) * 2020-09-23 2022-03-31 Seelos Therapeutics, Inc. Compositions and methods for inhibiting alpha-synuclein aggregation
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
CN115029360B (zh) * 2022-05-30 2024-08-02 上海勉亦生物科技有限公司 用于治疗粘多糖贮积症iiia型的转基因表达盒
KR20250142386A (ko) * 2023-02-03 2025-09-30 얀센 파마슈티카 엔브이 파킨슨병에 사용하기 위한 유전자 요법 벡터
WO2025065011A1 (en) * 2023-09-22 2025-03-27 The Regents Of The University Of Colorado, A Body Corporate Gene therapy for diabetic retinopathy
CN119193701B (zh) * 2024-09-13 2025-07-15 暨南大学 帕金森动物模型的构建方法与特异性靶向Parkin基因的gRNA及其应用

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060171935A1 (en) * 2005-01-31 2006-08-03 Asa Abeliovich Protecting cell therapy for neurological disorders including Parkinson's disease
WO2012067970A2 (en) * 2010-11-11 2012-05-24 Ted M Dawson Transcriptional repression leading to parkinson's disease
JP2015523065A (ja) * 2012-05-30 2015-08-13 アムファーク アクチェンゲゼルシャフト 組換えマイナス鎖ｒｎａウイルスベクターを使用する異種タンパク質発現方法

Family Cites Families (97)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US4861719A (en) 1986-04-25 1989-08-29 Fred Hutchinson Cancer Research Center DNA constructs for retrovirus packaging cell lines
EP0377676B1 (en) 1987-09-17 1995-05-24 Massachusetts Institute Of Technology Human erythroid-specific transcriptional enhancer
US5278056A (en) 1988-02-05 1994-01-11 The Trustees Of Columbia University In The City Of New York Retroviral packaging cell lines and process of using same
US5670488A (en) 1992-12-03 1997-09-23 Genzyme Corporation Adenovirus vector for gene therapy
WO1992001070A1 (en) 1990-07-09 1992-01-23 The United States Of America, As Represented By The Secretary, U.S. Department Of Commerce High efficiency packaging of mutant adeno-associated virus using amber suppressions
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
JP3534749B2 (ja) 1991-08-20 2004-06-07 アメリカ合衆国 アデノウイルスが介在する胃腸管への遺伝子の輸送
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
US5356802A (en) 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease
US5487994A (en) 1992-04-03 1996-01-30 The Johns Hopkins University Insertion and deletion mutants of FokI restriction endonuclease
DE69434860T2 (de) 1993-02-22 2007-03-15 The Rockefeller University Herstellung von helfer-freien retroviren mit hohem titer mittels transienter transfektion
FR2705361B1 (fr) 1993-05-18 1995-08-04 Centre Nat Rech Scient Vecteurs viraux et utilisation en thérapie génique.
FR2712812B1 (fr) 1993-11-23 1996-02-09 Centre Nat Rech Scient Composition pour la production de produits thérapeutiques in vivo.
CA2187626C (en) 1994-04-13 2009-11-03 Michael G. Kaplitt Aav-mediated delivery of dna to cells of the nervous system
IL116816A (en) 1995-01-20 2003-05-29 Rhone Poulenc Rorer Sa Cell for the production of a defective recombinant adenovirus or an adeno-associated virus and the various uses thereof
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
US6004797A (en) 1995-11-09 1999-12-21 Avigen, Inc. Adenovirus helper-free recombinant AAV Virion production
US6027721A (en) 1996-05-20 2000-02-22 Cytotherapeutics, Inc. Device and method for encapsulated gene therapy
US6136597A (en) 1997-09-18 2000-10-24 The Salk Institute For Biological Studies RNA export element
US20020065236A1 (en) 1998-09-09 2002-05-30 Yew Nelson S. CpG reduced plasmids and viral vectors
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US7994278B1 (en) 1999-08-06 2011-08-09 Nobel Biosciences Llc Biologically active polypeptides derived from a novel early stage pregnancy factor designated maternin (MA)
ATE351916T1 (de) 1999-10-12 2007-02-15 Pasteur Institut Lentivirale triplex-dns, und vektoren und rekombinante zellen, die lentivirale triplex-dns enthalten
CA2406743A1 (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
US6627442B1 (en) 2000-08-31 2003-09-30 Virxsys Corporation Methods for stable transduction of cells with hiv-derived viral vectors
GB0024550D0 (https=) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
US7575924B2 (en) 2000-11-13 2009-08-18 Research Development Foundation Methods and compositions relating to improved lentiviral vectors and their applications
EP1412493B1 (en) 2001-08-02 2011-10-05 Institut Clayton De La Recherche Methods and compositions relating to improved lentiviral vector production systems
EP1438075A4 (en) 2001-10-02 2006-04-19 Inst Clayton De La Rech METHOD AND COMPOSITIONS ASSOCIATED WITH LENTIVIRAL VECTORS WITH LIMITED EXPRESSION AND ITS APPLICATIONS
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
NZ536899A (en) * 2002-05-02 2008-04-30 Univ Rochester Vectors having both isoforms of beta-hexosaminidase
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
EP1529105B1 (en) 2002-08-12 2012-10-03 Société de commercialisation des produits de la recherche appliquée SOCPRA Sciences Santé et Humaines s.e.c. Methods to reprogram splice site selection in pre-messenger rnas
WO2004094642A2 (en) 2003-04-24 2004-11-04 Fondazione Centro San Raffaele Del Monte Tabor Lentiviral vectors carrying synthetic bi-directional promoters and uses thereof
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
CN1856576B (zh) 2003-09-30 2011-05-04 宾夕法尼亚州立大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
JP2007054069A (ja) 2005-08-24 2007-03-08 Medizinische Hochschule Hannover 自己不活性化レトロウイルスベクター
US7867484B2 (en) 2006-01-27 2011-01-11 University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
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
EP2152889B1 (en) 2007-05-29 2017-03-15 Nature Technology Corporation Vectors and methods for genetic immunization
WO2009012176A2 (en) 2007-07-14 2009-01-22 The University Of Iowa Research Foundation Methods and compositions for treating brain diseases
WO2009026116A2 (en) 2007-08-16 2009-02-26 Genizon Biosciences, Inc. Genemap of the human genes associated with longevity
AU2010241864B2 (en) 2009-04-30 2014-02-20 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Inducible interleukin-12
DE102009021592A1 (de) 2009-05-15 2010-11-18 Medizinische Hochschule Hannover ASLV-Vektorsystem
WO2011053825A2 (en) 2009-10-30 2011-05-05 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Compositions and methods for the treatment or prevention of mitochondrial diseases
JP2013523776A (ja) 2010-04-02 2013-06-17 セレジーン インコーポレイテッド パーキンソン病および脳のドーパミン作動性ニューロンのその他の障害を処置する方法
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
AU2013221212B2 (en) 2012-02-17 2018-08-09 The Children's Hospital Of Philadelphia AAV vector compositions and methods for gene transfer to cells, organs and tissues
AU2013243948A1 (en) * 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
KR102530118B1 (ko) 2012-07-25 2023-05-08 더 브로드 인스티튜트, 인코퍼레이티드 유도 ｄｎａ 결합 단백질 및 게놈 교란 도구 및 이의 적용
EP2792742A1 (en) 2013-04-17 2014-10-22 Universitätsklinikum Hamburg-Eppendorf (UKE) Gene-therapy vectors for treating cardiomyopathy
US9585971B2 (en) 2013-09-13 2017-03-07 California Institute Of Technology Recombinant AAV capsid protein
GB201318347D0 (en) 2013-10-16 2013-11-27 Ucl Business Plc Retroviral vectors
CN120174012A (zh) * 2013-10-24 2025-06-20 优尼科Ip有限公司 用于基因治疗神经疾病的aav-5假型载体
EP3760719A1 (en) 2013-11-18 2021-01-06 CRISPR Therapeutics AG Crispr-cas system materials and methods
CA2945335A1 (en) 2014-04-18 2015-10-22 Editas Medicine, Inc. Crispr-cas-related methods, compositions and components for cancer immunotherapy
CN107405411A (zh) 2014-05-01 2017-11-28 华盛顿大学 使用腺病毒载体的体内基因改造
IL248102B (en) 2014-05-02 2022-07-01 Genzyme Corp aav vectors for gene therapy of the central nervous system and retina
US11030531B2 (en) 2014-06-06 2021-06-08 Trustees Of Boston University DNA recombinase circuits for logical control of gene expression
US10781459B2 (en) 2014-06-20 2020-09-22 University Of Florida Research Foundation, Incorporated Methods of packaging multiple adeno-associated virus vectors
MA41346A (fr) 2015-01-12 2017-11-21 Juno Therapeutics Inc Eléments régulateurs post-transcriptionnels d'hépatite modifiée
US10350245B2 (en) 2015-01-21 2019-07-16 Fred Hutchinson Cancer Research Center Point-of-care and/or portable platform for gene therapy
CN107635575A (zh) 2015-03-10 2018-01-26 纽约市哥伦比亚大学理事会 重组glut1腺相关病毒载体构建体以及用于恢复glut1表达的相关方法
EA201792500A1 (ru) 2015-05-13 2018-04-30 Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания Aav-опосредованная экспрессия антител против гриппа и способы их использования
US20170029798A1 (en) 2015-07-27 2017-02-02 Cellivery Therapeutics, Inc. Development of Improved Cell-Permeable (iCP) Parkin Recombinant Protein as a Protein-Based Anti-Neurodegenerative Agent for the Treatment of Parkinson's Disease-Associated Phenotypes by Utilizing BBB-Penetrating Protein Delivery System MITT, Enabled by Advanced Macromolecule Transduction Domain (aMTD)
WO2017083750A1 (en) 2015-11-11 2017-05-18 Intrexon Corporation Compositions and methods for expression of multiple biologically active polypeptides from a single vector for treatment of cardiac conditions and other pathologies
KR102423442B1 (ko) 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
DK3405215T3 (da) 2016-01-19 2022-09-19 Univ California Fremgangsmåder til behandling af danon-sygdom og andre autofagilidelser
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
CA3021572A1 (en) 2016-04-20 2017-10-26 Centro De Investigaciones Energeticas, Medioambientales Y Tecnologicas Compositions and methods for enhanced gene expression of pklr
US11260081B2 (en) * 2016-06-29 2022-03-01 Ramot At Tel-Aviv University Ltd. Engineered parasites for delivering protein to the central nervous system (CNS)
CN110536966A (zh) 2016-09-08 2019-12-03 能源环境和技术研究中心O.A.,M.P. 用于范可尼贫血患者的基因疗法
JP7059285B2 (ja) 2016-09-30 2022-04-25 エステベ プアルマセウティカルス， エッセ．ア． ムコ多糖症の治療のためのアデノ随伴ウイルスベクター
US20180326022A1 (en) 2017-04-21 2018-11-15 The Trustees Of The University Of Pennsylvania Compositions and methods for improving heart function and treating heart failure
US20200199534A1 (en) 2017-04-27 2020-06-25 Fred Hutchinson Cancer Research Center Therapeutic formulations containing cd34+ stem cells derived from negative selection
EP3697452A4 (en) 2017-10-16 2021-11-24 Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, O.A., M.P. LENTIVIRAL VECTORS FOR ADMINISTRATION OF PKLR TO TREAT PYRUVATE KINASE DEFECTS
BR112020020841A2 (pt) 2018-04-11 2021-01-19 Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus Composições e métodos para transplante de célulastronco
CA3096293A1 (en) 2018-04-27 2019-10-31 Rocket Pharmaceuticals, Ltd. Gene therapy for cns degeneration
AU2019258830B2 (en) 2018-04-27 2025-12-18 Universität Heidelberg Modified AAV capsid polypeptides for treatment of muscular diseases
WO2020014523A1 (en) 2018-07-12 2020-01-16 Rocket Pharmaceuticals, Ltd. Gene therapy vectors for treatment of danon disease
KR20210046004A (ko) 2018-07-30 2021-04-27 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. 조혈 세포의 유전자 변형 방법
EP3833745A1 (en) 2018-08-10 2021-06-16 REGENXBIO Inc. Scalable method for recombinant aav production
JP2021533831A (ja) 2018-08-16 2021-12-09 スペースクラフト セブン リミテッド ライアビリティ カンパニー ウイルスベクターのための生成方法
AU2020221842A1 (en) 2019-02-12 2021-08-12 Spacecraft Seven, Llc Gene therapy vectors for treatment of Danon Disease
CN113994008A (zh) 2019-05-23 2022-01-28 太空飞船七有限责任公司 用于婴儿恶性骨硬化病的基因治疗载体
IL297391A (en) 2020-04-20 2022-12-01 Tenaya Therapeutics Inc An adeno-associated virus with an engineered capsid
JP2023535121A (ja) 2020-05-20 2023-08-16 スペースクラフト セブン リミテッド ライアビリティ カンパニー 操作されたパーキン及びその使用
JP2023526449A (ja) 2020-05-21 2023-06-21 ニスノバイオ ジーティー，エルエルシー パーキンソン病を治療するための、増加した活性を有するパーキン変異体の遺伝子治療送達
WO2022017630A1 (en) 2020-07-23 2022-01-27 Ucl Business Ltd GENE THERAPY VECTOR FOR eEF1A2 AND USES THEREOF
BR112023001336A2 (pt) 2020-08-05 2023-02-14 Spacecraft Seven Llc Terapia gênica de csrp3 (proteína 3 rica em cisteína e glicina)
AU2021321412A1 (en) 2020-08-05 2023-04-06 Spacecraft Seven, Llc Adeno-associated viral vector for GLUT1 expression and uses thereof
JP2023538519A (ja) 2020-08-07 2023-09-08 スペースクラフト セブン リミテッド ライアビリティ カンパニー Ａａｖベクターを使用したプラコフィリン－２（ｐｋｐ２）遺伝子治療
CA3201247A1 (en) 2020-12-07 2022-06-16 Gaurav Shah Treatment of danon disease
US20250135040A1 (en) 2022-02-08 2025-05-01 Spacecraft Seven, Llc Adeno-associated viral vector for glut1 expression and uses thereof

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060171935A1 (en) * 2005-01-31 2006-08-03 Asa Abeliovich Protecting cell therapy for neurological disorders including Parkinson's disease
WO2012067970A2 (en) * 2010-11-11 2012-05-24 Ted M Dawson Transcriptional repression leading to parkinson's disease
JP2015523065A (ja) * 2012-05-30 2015-08-13 アムファーク アクチェンゲゼルシャフト 組換えマイナス鎖ｒｎａウイルスベクターを使用する異種タンパク質発現方法

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
CURRENT OPINION IN STRUCTURAL BIOLOGY, vol. 38, JPN6023002136, 2016, pages 155 - 162, ISSN: 0005307669 *
MOCHIZUKI, HIDEKI ET AL., PARKINSONISM & RELATED DISORDERS, vol. 12, JPN6023002137, 2006, pages 107 - 109, ISSN: 0005307668 *

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