JP2017509632A5 - - Google Patents

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JP2017509632A5
JP2017509632A5 JP2016557218A JP2016557218A JP2017509632A5 JP 2017509632 A5 JP2017509632 A5 JP 2017509632A5 JP 2016557218 A JP2016557218 A JP 2016557218A JP 2016557218 A JP2016557218 A JP 2016557218A JP 2017509632 A5 JP2017509632 A5 JP 2017509632A5
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Japan
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nucleic acid
raav
seq
acid encoding
mir
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JP2016557218A
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Japanese (ja)
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JP6669664B2 (ja
JP2017509632A (ja
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Priority claimed from PCT/US2015/021896 external-priority patent/WO2015143418A2/en
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JP2016557218A 2014-03-21 2015-03-20 網膜色素変性症のための遺伝子治療 Active JP6669664B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201461969027P 2014-03-21 2014-03-21
US61/969,027 2014-03-21
PCT/US2015/021896 WO2015143418A2 (en) 2014-03-21 2015-03-20 Gene therapy for retinitis pigmentosa

Related Child Applications (1)

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JP2019224202A Division JP7048563B2 (ja) 2014-03-21 2019-12-12 網膜色素変性症のための遺伝子治療

Publications (3)

Publication Number Publication Date
JP2017509632A JP2017509632A (ja) 2017-04-06
JP2017509632A5 true JP2017509632A5 (enExample) 2018-04-26
JP6669664B2 JP6669664B2 (ja) 2020-03-18

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JP2016557218A Active JP6669664B2 (ja) 2014-03-21 2015-03-20 網膜色素変性症のための遺伝子治療
JP2019224202A Active JP7048563B2 (ja) 2014-03-21 2019-12-12 網膜色素変性症のための遺伝子治療
JP2022046280A Active JP7534348B2 (ja) 2014-03-21 2022-03-23 網膜色素変性症のための遺伝子治療

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JP2019224202A Active JP7048563B2 (ja) 2014-03-21 2019-12-12 網膜色素変性症のための遺伝子治療
JP2022046280A Active JP7534348B2 (ja) 2014-03-21 2022-03-23 網膜色素変性症のための遺伝子治療

Country Status (36)

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US (3) US10383953B2 (enExample)
EP (3) EP3628334B1 (enExample)
JP (3) JP6669664B2 (enExample)
KR (3) KR20230006039A (enExample)
CN (2) CN115252823A (enExample)
AR (1) AR099837A1 (enExample)
AU (3) AU2015230942B2 (enExample)
CA (2) CA2943185C (enExample)
CL (1) CL2016002333A1 (enExample)
CR (1) CR20160480A (enExample)
DK (2) DK3628334T5 (enExample)
DO (1) DOP2016000237A (enExample)
EA (1) EA201691891A1 (enExample)
EC (1) ECSP16083000A (enExample)
ES (2) ES2957840T3 (enExample)
FI (1) FI3628334T3 (enExample)
HR (2) HRP20192141T1 (enExample)
HU (2) HUE046454T2 (enExample)
IL (3) IL247543B (enExample)
LT (2) LT3628334T (enExample)
MA (1) MA39390B2 (enExample)
MX (2) MX376190B (enExample)
MY (2) MY190726A (enExample)
NZ (1) NZ724622A (enExample)
PE (1) PE20161252A1 (enExample)
PH (1) PH12016501684B1 (enExample)
PL (2) PL3119437T3 (enExample)
PT (2) PT3628334T (enExample)
RS (2) RS59634B1 (enExample)
SG (3) SG10201912968WA (enExample)
SI (2) SI3628334T1 (enExample)
TW (2) TWI780401B (enExample)
UA (1) UA120050C2 (enExample)
UY (1) UY36044A (enExample)
WO (1) WO2015143418A2 (enExample)
ZA (1) ZA201605924B (enExample)

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115252823A (zh) * 2014-03-21 2022-11-01 建新公司 用于色素性视网膜炎的基因疗法
JP2018506304A (ja) 2015-02-10 2018-03-08 ジェンザイム・コーポレーション バリアントＲＮＡｉ
CN108472390B (zh) * 2015-12-03 2022-04-15 弗里德里克·米谢尔生物医学研究所 SynP162,用于基因在视杆光感受器中特异性表达的启动子
JP6969798B2 (ja) 2015-12-03 2021-11-24 フリードリッヒ ミーシェー インスティトゥート フォー バイオメディカル リサーチ ＳｙｎＰ１６０、桿体光受容器における遺伝子の特異的発現のためのプロモーター
US11390866B2 (en) 2015-12-04 2022-07-19 Sorbonne Université Promoters and uses thereof
IL261014B2 (en) * 2016-03-01 2023-10-01 Univ Florida Aav vectors for treatment of dominant retinitis pigmentosa
KR20190039703A (ko) * 2016-07-05 2019-04-15 더 존스 홉킨스 유니버시티 Crispr/cas9-기반 조성물 및 망막 변성을 치료하기 위한 방법
JP7257377B2 (ja) * 2017-03-21 2023-04-13 ミヒャラキス，スティリアノス Ｃｎｇｂ１連鎖性網膜色素変性症の治療のための遺伝子療法
AR113134A1 (es) 2017-09-22 2020-01-29 Genzyme Corp Arni variante
WO2019067766A1 (en) * 2017-09-27 2019-04-04 Sigilon Therapeutics, Inc. METHODS, COMPOSITIONS AND IMPLANTABLE ELEMENTS COMPRISING ACTIVE CELLS
CN107961380B (zh) * 2017-11-23 2020-05-05 清华大学 试剂在制备药物中的用途、筛选药物的方法以及药物组合物
US12162994B2 (en) 2018-03-02 2024-12-10 Sigilon Therapeutics, Inc. Biocompatible hydrogel capsules and process for preparing same
US20210017509A1 (en) * 2018-03-23 2021-01-21 The Trustees Of Columbia University In The City Of New York Gene Editing for Autosomal Dominant Diseases
CN112203646A (zh) 2018-04-04 2021-01-08 西吉隆医疗股份有限公司 可植入颗粒和相关方法
CA3102095A1 (en) * 2018-06-01 2019-12-05 University Of Florida Research Foundation, Incorporated Compositions and methods for treatment of dominant retinitis pigmentosa
GB201817469D0 (en) * 2018-10-26 2018-12-12 Univ Oxford Innovation Ltd Gene therapy for retinal disease
CN111518813B (zh) * 2019-02-03 2023-04-28 武汉纽福斯生物科技有限公司 视紫红质的编码序列、其表达载体构建及其应用
AU2020232692A1 (en) 2019-03-04 2021-09-30 The Trustees Of The University Of Pennsylvania Neuroprotective gene therapy targeting the AKT pathway
EP3941530A4 (en) * 2019-03-21 2022-12-14 PTC Therapeutics, Inc. ANGELMAN SYNDROME TREATMENT VECTOR AND PROCEDURES
WO2021030745A1 (en) * 2019-08-15 2021-02-18 The Children's Hospital Of Philadelphia Combined transgene and intron-derived mirna therapy for treatment of sca1
WO2021142447A1 (en) * 2020-01-10 2021-07-15 Solid Biosciences Inc. Viral vector for combination therapy
MX2022009252A (es) * 2020-01-29 2022-11-09 Genzyme Corp Proteinas de la capside de virus adeno-asociados modificadas para la terapia genica ocular y metodos de uso de las mismas.
KR102396200B1 (ko) * 2020-07-24 2022-05-12 알지노믹스 주식회사 로돕신 전사체에 특이적인 트랜스-스플라이싱 라이보자임 및 이의 용도
JP2023535956A (ja) * 2020-07-29 2023-08-22 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド 改善されたｘ連鎖性網膜分離症のａａｖ媒介療法
WO2022221405A1 (en) * 2021-04-16 2022-10-20 Regeneron Pharmaceuticals, Inc. Treatment of x-linked juvenile retinoschisis
TWI832531B (zh) * 2022-11-02 2024-02-11 慈濟學校財團法人慈濟大學 Rip1抑制劑或mlkl抑制劑用於治療或預防遺傳性視網膜失養症的用途
CN117625619B (zh) * 2023-12-05 2024-11-12 云舟生物科技(广州)股份有限公司 核酸分子及其作为特异启动子的应用
CN120230798A (zh) * 2023-12-29 2025-07-01 上海朗昇生物科技有限公司 用于视网膜基因递送的重组腺相关病毒载体及其应用

Family Cites Families (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5262529A (en) * 1990-01-24 1993-11-16 President And Fellows Of Harvard College Diagnosis of hereditary retinal degenerative diseases
US5985583A (en) * 1992-06-23 1999-11-16 Mount Sinai School Of Medicine Of The City University Of New York Cloning and expression of gonadotropin-releasing hormone receptor
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
ES2308989T3 (es) 1999-08-09 2008-12-16 Targeted Genetics Corporation Aumento de la expresion de una secuencia nucleotidica heterologa a partir de vectores viricos recombinantes que contienen una secuencia que forman pares de bases intracatenarios.
ES2505700T3 (es) 2000-06-01 2014-10-10 University Of North Carolina At Chapel Hill Vectores de parvovirus duplicados
IL161827A0 (en) 2001-11-13 2005-11-20 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novelsequences identified thereby
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
WO2005071092A1 (ja) * 2004-01-22 2005-08-04 Dnavec Research Inc. サイトメガロウイルスエンハンサーおよびニワトリβ-アクチンプロモーターを含むハイブリッドプロモーターを利用したマイナス鎖RNAウイルスベクターの製造方法
US7765583B2 (en) 2005-02-28 2010-07-27 France Telecom System and method for managing virtual user domains
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
WO2007120542A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid library and aav capsid proteins
WO2008125846A2 (en) * 2007-04-12 2008-10-23 The Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth Genetic suppression and replacement
US20090214478A1 (en) * 2008-02-21 2009-08-27 Alberto Auricchio Method of treating ocular diseases by gene therapy
US9217155B2 (en) * 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US8734809B2 (en) * 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
US9078914B2 (en) 2009-09-10 2015-07-14 Velin-Pharma A/S Method for the preparation of micro-RNA and its therapeutic application
ES2680915T3 (es) 2010-01-28 2018-09-11 The Children's Hospital Of Philadelphia Plataforma de fabricación escalable para la purificación de vectores virales y vectores virales purificados de este modo para su utilización en terapia génica
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
WO2012057363A1 (ja) * 2010-10-27 2012-05-03 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
JP6224459B2 (ja) 2011-02-17 2017-11-01 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組織特異性を改変し、ａａｖ９媒介遺伝子導入を改善するための組成物および方法
JP5884152B2 (ja) 2011-07-29 2016-03-15 シャープ株式会社 基地局、端末、通信システムおよび通信方法
ITRM20110685A1 (it) * 2011-12-23 2013-06-24 Internat Ct For Genetic En Gineering And Microrna per la rigenerazione cardiaca attraverso l induzione della proliferazione dei miociti cardiaci
KR102057540B1 (ko) * 2012-02-17 2019-12-19 더 칠드런스 호스피탈 오브 필라델피아 세포, 기관 및 조직으로의 유전자 전이를 위한 ａａｖ 벡터 조성물 및 방법
WO2013151665A2 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
US9127274B2 (en) * 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
EP4570908A3 (en) * 2012-05-25 2025-10-22 The Regents of the University of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
CN105120901A (zh) * 2012-07-11 2015-12-02 宾夕法尼亚大学托管会 Rpgr x染色体连锁视网膜退化的aav介导的基因治疗
US9512425B2 (en) * 2012-10-23 2016-12-06 Cornell University Inhibiting migration of cancer cells
US9884125B2 (en) 2013-10-04 2018-02-06 Merck Sharp & Dohme Corp. Glucose-responsive insulin conjugates
CN115252823A (zh) 2014-03-21 2022-11-01 建新公司 用于色素性视网膜炎的基因疗法

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