JP6994140B2 - 非ウイルス的遺伝子導入のための閉鎖型直鎖状二重鎖dna - Google Patents

非ウイルス的遺伝子導入のための閉鎖型直鎖状二重鎖dna Download PDF

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JP6994140B2
JP6994140B2 JP2018546429A JP2018546429A JP6994140B2 JP 6994140 B2 JP6994140 B2 JP 6994140B2 JP 2018546429 A JP2018546429 A JP 2018546429A JP 2018546429 A JP2018546429 A JP 2018546429A JP 6994140 B2 JP6994140 B2 JP 6994140B2
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nucleic acid
sequence
cedna
protein
cell
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JP2019506889A (ja
JP2019506889A5 (cg-RX-API-DMAC7.html
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コーチン,ロバート,エム.
チェッキーニ,シルヴァン
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Voyager Therapeutics Inc
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JP2018546429A 2016-03-03 2017-03-03 非ウイルス的遺伝子導入のための閉鎖型直鎖状二重鎖dna Active JP6994140B2 (ja)

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US201662303047P 2016-03-03 2016-03-03
US62/303,047 2016-03-03
US201662394720P 2016-09-14 2016-09-14
US62/394,720 2016-09-14
US201662406913P 2016-10-11 2016-10-11
US62/406,913 2016-10-11
PCT/US2017/020828 WO2017152149A1 (en) 2016-03-03 2017-03-03 Closed-ended linear duplex dna for non-viral gene transfer

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JP2022191114A Pending JP2023024489A (ja) 2016-03-03 2022-11-30 非ウイルス的遺伝子導入のための閉鎖型直鎖状二重鎖dna

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JP2022191114A Pending JP2023024489A (ja) 2016-03-03 2022-11-30 非ウイルス的遺伝子導入のための閉鎖型直鎖状二重鎖dna

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Families Citing this family (55)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
DK3218386T3 (da) 2014-11-14 2021-06-07 Voyager Therapeutics Inc Modulatorisk polynukleotid
CA3193811A1 (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
RU2752882C2 (ru) 2016-03-03 2021-08-11 Юниверсити Оф Массачусетс Линейная дуплексная днк с закрытым концом для невирусного переноса генов
AU2017267665C1 (en) 2016-05-18 2023-10-05 Voyager Therapeutics, Inc. Modulatory polynucleotides
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
KR102780287B1 (ko) 2017-08-09 2025-03-14 바이오버라티브 테라퓨틱스 인크. 핵산 분자 및 이의 용도
MX2020002501A (es) * 2017-09-08 2020-09-17 Generation Bio Co Formulaciones de nanoparticulas lipidas de vectores de adn libres de capsidos, no virales.
KR20200051011A (ko) * 2017-09-08 2020-05-12 제너레이션 바이오 컴퍼니 변형된 폐쇄-종결된 dna(cedna)
IL273448B2 (en) 2017-09-20 2025-05-01 Univ California A gene therapy strategy to restore electrical and cardiac function, and cardiac structure, in arrhythmogenic right ventricular cardiomyopathy
US12098385B2 (en) 2017-09-20 2024-09-24 The Regents Of The University Of California Gene therapy strategy to restore cardiac electrical and structural function in arrhythmogenic right ventricular cardiomyopathy
WO2019079362A1 (en) * 2017-10-16 2019-04-25 Massachusetts Institute Of Technology HOST-PATHOGENIC INTERACTION OF MYCOBACTERIUM TUBERCULOSIS
AU2018378672A1 (en) * 2017-12-06 2020-07-09 Generation Bio Co. Gene editing using a modified closed-ended dna (ceDNA)
US20210371877A1 (en) * 2017-12-18 2021-12-02 Spark Therapeutics, Inc. Adeno-associated virus (aav) vector lipid nanoparticle compositions and methods of use
SG11202005271TA (en) 2018-01-19 2020-07-29 Generation Bio Co Closed-ended dna vectors obtainable from cell-free synthesis and process for obtaining cedna vectors
CN111818942A (zh) * 2018-02-14 2020-10-23 世代生物公司 非病毒dna载体以及其用于产生抗体和融合蛋白的用途
CA3092059A1 (en) * 2018-02-22 2019-08-29 Generation Bio Co. Controlled expression of transgenes using close-ended dna (cedna) vectors
MA52116A (fr) * 2018-03-02 2021-01-06 Generation Bio Co Vecteurs d'adn à extrémité fermée (cedna) pour l'insertion de transgènes au niveau de havres génomiques sécuritaires (gsh) dans des génomes humains et murins
CA3092088A1 (en) * 2018-03-15 2019-09-19 Igtx, Llc Synthetic dna vectors and methods of use
WO2019191109A1 (en) * 2018-03-28 2019-10-03 The Board Of Trustees Of The Leland Stanford Junior University Microrna inhibitors and their use in treating epiretinal membranes
US12319929B2 (en) 2018-05-15 2025-06-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of Parkinson's disease
WO2019226650A1 (en) 2018-05-23 2019-11-28 Modernatx, Inc. Delivery of dna
WO2019237383A1 (zh) * 2018-06-16 2019-12-19 深圳市博奥康生物科技有限公司 用于人 tnfsf18 基因编辑的修饰载体、其制备方法及应用
WO2019237391A1 (zh) * 2018-06-16 2019-12-19 深圳市博奥康生物科技有限公司 CRISPR/Cas9 靶向敲除人 TXGP1 基因及其特异性 gRNA
EP3810782A2 (en) * 2018-06-22 2021-04-28 Asklepios Biopharmaceutical, Inc. Vectors for gene delivery that persist within cells
BR112021002017A2 (pt) 2018-08-09 2021-05-11 Bioverativ Therapeutics Inc. moléculas de ácido nucleico e usos das mesmas para terapia genética não viral
US20210310000A1 (en) * 2018-08-17 2021-10-07 City Of Hope Compounds of chemically modified oligonucleotides and methods of use thereof
CA3114621A1 (en) 2018-09-28 2020-04-02 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
BR112021007102A2 (pt) * 2018-11-09 2021-08-03 Generation Bio Co. dna com extremidade fechada modificada (cedna) que compreende repetições terminais invertidas modificadas simétricas
KR20210119416A (ko) * 2019-01-24 2021-10-05 제너레이션 바이오 컴퍼니 폐쇄-말단 dna (cedna), 및 유전자 또는 핵산 치료 관련 면역 반응을 감소시키는 방법에서의 이의 용도
WO2020168222A1 (en) * 2019-02-15 2020-08-20 Generation Bio Co. Modulation of rep protein activity in closed-ended dna (cedna) production
MA55223A (fr) * 2019-03-06 2022-01-12 Generation Bio Co Adn à extrémité fermée (cedna) et composés de modulation immunitaire
SG11202109426YA (en) * 2019-03-06 2021-09-29 Generation Bio Co Non-active lipid nanoparticles with non-viral, capsid free dna
EP3938515A4 (en) * 2019-03-13 2022-12-28 Generation Bio Co. NON-VIRAL DNA VECTORS AND THEIR USES FOR EXPRESSING PHENYLALANINE HYDROXYLASE (PAH) THERAPEUTIC AGENTS
KR20210151785A (ko) * 2019-03-13 2021-12-14 제너레이션 바이오 컴퍼니 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도
GB2617512B (en) * 2019-04-05 2023-12-27 Earli Inc Improved methods and compositions for synthetic biomarkers
CA3151464A1 (en) 2019-09-18 2021-03-25 Bruce C. SCHNEPP Synthetic dna vectors and methods of use
EP4110931A4 (en) 2020-02-25 2024-03-27 University of Massachusetts Inducible single aav system and uses thereof
AU2021241696A1 (en) 2020-03-27 2022-12-01 Generation Bio Co. Novel lipids and nanoparticle compositions thereof
US20210330759A1 (en) * 2020-04-22 2021-10-28 The Cleveland Clinic Foundation Methods and compositions for treating cytokine storm, ards, and acute lung injury using beta-glucocerebrosidase
AU2021285812A1 (en) 2020-06-01 2023-01-05 Modernatx, Inc. Phenylalanine hydroxylase variants and uses thereof
CA3189740A1 (en) 2020-07-27 2022-02-03 Anjarium Biosciences Ag Compositions of dna molecules, methods of making therefor, and methods of use thereof
CN116194152A (zh) 2020-08-23 2023-05-30 比奥维拉迪维治疗股份有限公司 用于改进封闭端DNA(ceDNA)的产生的经修饰的杆状病毒系统
AU2021342503A1 (en) * 2020-09-16 2023-04-06 Generation Bio Co. Non-viral DNA vectors and uses thereof for expressing FVIII therapeutics
KR20240005903A (ko) * 2021-05-07 2024-01-12 제너레이션 바이오 컴퍼니 백신 전달을 위한 비바이러스성 dna 벡터
CA3218126A1 (en) * 2021-05-07 2022-11-10 Matthew MANGANIELLO Lyophilized non-viral dna vector compositions and uses thereof
US20240263195A1 (en) 2021-05-11 2024-08-08 Modernatx, Inc. Non-viral delivery of dna for prolonged polypeptide expression in vivo
WO2023028004A1 (en) * 2021-08-23 2023-03-02 Avirmax, Inc. Compositions and methods for transgene expression
JP2024534123A (ja) 2021-08-23 2024-09-18 バイオベラティブ セラピューティクス インコーポレイテッド バキュロウイルス発現システム
CN114525282A (zh) * 2022-03-04 2022-05-24 西南大学 一种二价核酸适配体及其设计方法
IL322201A (en) * 2023-02-17 2025-09-01 Flagship Pioneering Innovations Vii Llc DNA structures containing modified uracil
WO2024197242A1 (en) 2023-03-23 2024-09-26 Carbon Biosciences, Inc. Protoparvovirus compositions comprising a protoparvovirus variant vp1 capsid polypeptide and related methods
WO2024249438A1 (en) * 2023-05-26 2024-12-05 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Copiparvovirus genetic vector platform
WO2025212926A1 (en) * 2024-04-05 2025-10-09 University Of Massachusetts Aav-mediated rhoa knockdown

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004508041A (ja) 2000-09-08 2004-03-18 メディジーン・アクチェンゲゼルシャフト 組換え型アデノ随伴ウイルス(rAAV)をパッケージングする親細胞、それらの生成方法、およびその使用

Family Cites Families (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US5549910A (en) 1989-03-31 1996-08-27 The Regents Of The University Of California Preparation of liposome and lipid complex compositions
US5252334A (en) 1989-09-08 1993-10-12 Cygnus Therapeutic Systems Solid matrix system for transdermal drug delivery
US5316908A (en) 1990-07-13 1994-05-31 Life Technologies, Inc. Size markers for electrophoretic analysis of DNA
JP3218637B2 (ja) 1990-07-26 2001-10-15 大正製薬株式会社 安定なリポソーム水懸濁液
JP2958076B2 (ja) 1990-08-27 1999-10-06 株式会社ビタミン研究所 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法
US5399363A (en) 1991-01-25 1995-03-21 Eastman Kodak Company Surface modified anticancer nanoparticles
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
AU689815B2 (en) * 1993-08-30 1998-04-09 Promega Corporation Nucleic acid purification compositions and methods
US5741516A (en) 1994-06-20 1998-04-21 Inex Pharmaceuticals Corporation Sphingosomes for enhanced drug delivery
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5697899A (en) 1995-02-07 1997-12-16 Gensia Feedback controlled drug delivery system
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US5738868A (en) 1995-07-18 1998-04-14 Lipogenics Ltd. Liposome compositions and kits therefor
US5656016A (en) 1996-03-18 1997-08-12 Abbott Laboratories Sonophoretic drug delivery system
US5797898A (en) 1996-07-02 1998-08-25 Massachusetts Institute Of Technology Microchip drug delivery devices
US5783208A (en) 1996-07-19 1998-07-21 Theratech, Inc. Transdermal drug delivery matrix for coadministering estradiol and another steroid
US7098320B1 (en) 1996-07-29 2006-08-29 Nanosphere, Inc. Nanoparticles having oligonucleotides attached thereto and uses therefor
US5779708A (en) 1996-08-15 1998-07-14 Cyberdent, Inc. Intraosseous drug delivery device and method
IL128780A0 (en) 1996-09-06 2000-01-31 Univ Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase
US6177403B1 (en) 1996-10-21 2001-01-23 The Trustees Of The University Of Pennsylvania Compositions, methods, and apparatus for delivery of a macromolecular assembly to an extravascular tissue of an animal
AU1750400A (en) 1998-12-04 2000-06-26 Mosaic Technologies, Inc. Method for the immobilization of oligonucleotides
US8241622B2 (en) * 2001-07-13 2012-08-14 University Of Iowa Research Foundation Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
KR101133799B1 (ko) 2006-08-18 2012-04-24 에프. 호프만-라 로슈 아게 폴리뉴클레오타이드의 생체내 전달을 위한 다가접합체
CA2927045A1 (en) 2006-10-03 2008-04-10 Muthiah Manoharan Lipid containing formulations
WO2009073809A2 (en) 2007-12-04 2009-06-11 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
US8507455B2 (en) 2007-12-04 2013-08-13 Alnylam Pharmaceuticals, Inc. Folate conjugates
LT2440183T (lt) 2009-06-10 2018-08-10 Arbutus Biopharma Corporation Patobulinta lipido kompozicija
WO2012068187A1 (en) 2010-11-19 2012-05-24 Merck Sharp & Dohme Corp. Poly(amide) polymers for the delivery of oligonucleotides
EP2500434A1 (en) * 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
RU2752882C2 (ru) 2016-03-03 2021-08-11 Юниверсити Оф Массачусетс Линейная дуплексная днк с закрытым концом для невирусного переноса генов

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004508041A (ja) 2000-09-08 2004-03-18 メディジーン・アクチェンゲゼルシャフト 組換え型アデノ随伴ウイルス(rAAV)をパッケージングする親細胞、それらの生成方法、およびその使用

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Virology Journal,2005年,vol.2, no.43,p.1-17

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