CN104619832B - 经修饰的腺伴随病毒载体组合物 - Google Patents

经修饰的腺伴随病毒载体组合物 Download PDF

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CN104619832B
CN104619832B CN201380035802.XA CN201380035802A CN104619832B CN 104619832 B CN104619832 B CN 104619832B CN 201380035802 A CN201380035802 A CN 201380035802A CN 104619832 B CN104619832 B CN 104619832B
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aav
vector
sequence
nucleic acid
stuffer
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CN104619832A (zh
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B.L.戴维森
M.希尔
R.布德罗
A.M.蒙特斯
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University of Iowa Research Foundation UIRF
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    • 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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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CN201380035802.XA 2012-07-06 2013-03-14 经修饰的腺伴随病毒载体组合物 Active CN104619832B (zh)

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US201261668839P 2012-07-06 2012-07-06
US61/668839 2012-07-06
PCT/US2013/031644 WO2014007858A1 (en) 2012-07-06 2013-03-14 Modified adeno-associated virus vector compositions

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CN104619832B true CN104619832B (zh) 2021-03-16

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US (2) US9540659B2 (https=)
EP (1) EP2870237B1 (https=)
JP (1) JP6366581B2 (https=)
CN (1) CN104619832B (https=)
AU (1) AU2013287261B2 (https=)
BR (1) BR112015000161A2 (https=)
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ES (1) ES2703177T3 (https=)
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IN (1) IN2015KN00074A (https=)
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Families Citing this family (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2679843C2 (ru) * 2012-07-06 2019-02-13 Юниверсити Оф Айова Рисерч Фаундейшн Векторные композиции с модифицированным аденоассоциированным вирусом
EP3146051B8 (en) * 2014-05-20 2019-11-27 University of Iowa Research Foundation Huntington's disease therapeutic compounds
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
RU2716991C2 (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
US20190000940A1 (en) * 2015-07-31 2019-01-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of aadc deficiency
WO2017161273A1 (en) * 2016-03-18 2017-09-21 The Children's Hospital Of Philadelphia Therapeutic for treatment of diseases including the central nervous system
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
KR102427379B1 (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
WO2018044933A1 (en) 2016-08-30 2018-03-08 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
JP2020518258A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法
JP2020518259A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ハンチントン病治療組成物および方法
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
AU2018338728B2 (en) 2017-09-29 2025-01-02 Centre National De La Recherche Scientifique (Cnrs) Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
EP3697908A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
EP3724326A1 (en) * 2017-12-11 2020-10-21 Editas Medicine, Inc. Cpf1-related methods and compositions for gene editing
US20190256867A1 (en) 2018-02-01 2019-08-22 Homology Medicines, Inc. Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
JP7244547B2 (ja) 2018-02-19 2023-03-22 ホモロジー・メディシンズ・インコーポレイテッド Ｆ８遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
EP3774852A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
AU2019247748A1 (en) 2018-04-03 2020-10-08 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
KR20210019996A (ko) 2018-05-15 2021-02-23 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 조성물 및 방법
US20210254103A1 (en) * 2018-07-02 2021-08-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
CA3140049A1 (en) 2019-05-14 2020-11-19 Duke University Compositions and methods for the treatment of atpase-mediated diseases
EP3999647A1 (en) * 2019-07-18 2022-05-25 Lysogene Compositions and methods for the treatment of sanfilippo disease and other disorders
JP2022551739A (ja) 2019-10-17 2022-12-13 ストライドバイオ，インコーポレイテッド ニーマン・ピック病ｃ型の治療のためのアデノ随伴ウイルスベクター
US12611436B2 (en) 2019-10-17 2026-04-28 Sarepta Therapeutics, Inc. AAV transfer cassette
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
CN115768900A (zh) * 2020-05-11 2023-03-07 戈迪安生物技术公司 病毒递送媒剂选择
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
AU2021328475A1 (en) 2020-08-19 2023-03-16 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
JP2024506296A (ja) * 2021-02-05 2024-02-13 メイズ セラピューティクス， インコーポレイテッド スタッファーポリヌクレオチド配列を含むベクター
US20240218397A1 (en) * 2021-05-04 2024-07-04 Regenxbio Inc. Novel aav vectors and methods and uses thereof
GB2623164A (en) * 2022-08-09 2024-04-10 Univ Sheffield Viral vector

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999061034A1 (en) * 1998-05-28 1999-12-02 The Regents Of The University Of Michigan Improved vectors
WO2011133874A1 (en) * 2010-04-23 2011-10-27 University Of Massachusetts Multicistronic expression constructs

Family Cites Families (10)

* 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
JPH1033175A (ja) * 1996-07-24 1998-02-10 Hisamitsu Pharmaceut Co Inc 組換えアデノ随伴ウイルスベクターの製造方法
US6943153B1 (en) * 1999-03-15 2005-09-13 The Regents Of The University Of California Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
WO2001032899A1 (en) * 1999-10-29 2001-05-10 Takara Shuzo Co., Ltd. Gene transfer method
US7132277B1 (en) * 2000-01-31 2006-11-07 Merck & Co., Inc. Helper dependent vector system for gene therapy
US7387896B2 (en) 2003-03-26 2008-06-17 The Regents Of The University Of Michigan MicroRNA vectors
BRPI0415563A (pt) * 2003-10-20 2007-01-02 Nsgene As método para o tratamento do mal de parkinson, uso de um vetor viral, vetor de expressão viral, composição farmacêutica, célula hospedeira isolada, linhagem de células de empacotamento, mamìfero não humano quimérico, dispositivo de cultura celular implantável, cápsula biocompatìvel, método para tratamento de uma doença do sistema nervoso, célula de mamìfero, e, método para produzir neurturin ou um seu equivalente funcional
WO2009134681A2 (en) * 2008-04-30 2009-11-05 The Trustees Of The University Of Pennsylvania Aav7 viral vectors for targeted delivery of rpe cells
WO2012109667A1 (en) 2011-02-12 2012-08-16 University Of Iowa Research Foundation Therapeutic compounds
RU2679843C2 (ru) * 2012-07-06 2019-02-13 Юниверсити Оф Айова Рисерч Фаундейшн Векторные композиции с модифицированным аденоассоциированным вирусом

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999061034A1 (en) * 1998-05-28 1999-12-02 The Regents Of The University Of Michigan Improved vectors
WO2011133874A1 (en) * 2010-04-23 2011-10-27 University Of Massachusetts Multicistronic expression constructs

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
"Adeno-Associated Virus Vectors for Short Hairpin RNA Expression";Dirk Grimm 等;《Methods in Enzymology》;20050112;第392卷(第2期);第381页第1段和第396页第2段-第397页第2段 *
"GenBank Accession No：AC005516.1";登录号;《GenBank》;19981121;ORIGIN部分 *

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RU2015103901A (ru) 2016-08-27
AU2013287261B2 (en) 2019-03-07
US9540659B2 (en) 2017-01-10
CN104619832A (zh) 2015-05-13
CA2878401A1 (en) 2014-01-09
EP2870237A1 (en) 2015-05-13
JP2015522280A (ja) 2015-08-06
AU2013287261A1 (en) 2015-01-22
US20150184197A1 (en) 2015-07-02
US20170306355A1 (en) 2017-10-26
BR112015000161A2 (pt) 2017-06-27
CA2878401C (en) 2022-07-19
JP6366581B2 (ja) 2018-08-08
WO2014007858A1 (en) 2014-01-09
IN2015KN00074A (https=) 2015-07-31
ES2703177T3 (es) 2019-03-07
EP2870237B1 (en) 2018-09-26
EP2870237A4 (en) 2015-12-23
RU2679843C2 (ru) 2019-02-13

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