JP2020527335A5 - - Google Patents

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Publication number
JP2020527335A5
JP2020527335A5 JP2019569784A JP2019569784A JP2020527335A5 JP 2020527335 A5 JP2020527335 A5 JP 2020527335A5 JP 2019569784 A JP2019569784 A JP 2019569784A JP 2019569784 A JP2019569784 A JP 2019569784A JP 2020527335 A5 JP2020527335 A5 JP 2020527335A5
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JP
Japan
Prior art keywords
promoter
aav
sequence
seq
vector according
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JP2019569784A
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English (en)
Japanese (ja)
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JP7766393B2 (ja
JP2020527335A (ja
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Priority claimed from PCT/US2018/037592 external-priority patent/WO2018232149A1/en
Publication of JP2020527335A publication Critical patent/JP2020527335A/ja
Publication of JP2020527335A5 publication Critical patent/JP2020527335A5/ja
Priority to JP2023098376A priority Critical patent/JP2023116709A/ja
Application granted granted Critical
Publication of JP7766393B2 publication Critical patent/JP7766393B2/ja
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JP2019569784A 2017-06-14 2018-06-14 眼疾患のための遺伝子療法 Active JP7766393B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023098376A JP2023116709A (ja) 2017-06-14 2023-06-15 眼疾患のための遺伝子療法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762519821P 2017-06-14 2017-06-14
US62/519,821 2017-06-14
PCT/US2018/037592 WO2018232149A1 (en) 2017-06-14 2018-06-14 Gene therapy for ocular disorders

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023098376A Division JP2023116709A (ja) 2017-06-14 2023-06-15 眼疾患のための遺伝子療法

Publications (3)

Publication Number Publication Date
JP2020527335A JP2020527335A (ja) 2020-09-10
JP2020527335A5 true JP2020527335A5 (enExample) 2021-07-26
JP7766393B2 JP7766393B2 (ja) 2025-11-10

Family

ID=64660849

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2019569784A Active JP7766393B2 (ja) 2017-06-14 2018-06-14 眼疾患のための遺伝子療法
JP2023098376A Pending JP2023116709A (ja) 2017-06-14 2023-06-15 眼疾患のための遺伝子療法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2023098376A Pending JP2023116709A (ja) 2017-06-14 2023-06-15 眼疾患のための遺伝子療法

Country Status (4)

Country Link
US (1) US11827898B2 (enExample)
EP (1) EP3638316A4 (enExample)
JP (2) JP7766393B2 (enExample)
WO (1) WO2018232149A1 (enExample)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11827898B2 (en) * 2017-06-14 2023-11-28 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
AU2020232692A1 (en) * 2019-03-04 2021-09-30 The Trustees Of The University Of Pennsylvania Neuroprotective gene therapy targeting the AKT pathway
CA3191545A1 (en) * 2020-09-02 2022-03-10 David H. Kirn Codon optimized rep1 genes and uses thereof
MX2023008826A (es) 2021-02-01 2023-09-15 Regenxbio Inc Terapia génica para lipofuscinosis neuronal ceroidea.
CN113322281B (zh) * 2021-05-12 2024-01-05 成都金唯科生物科技有限公司 一种高效组织特异性表达rs1蛋白的重组腺相关病毒及应用
EP4551596A4 (en) * 2022-07-06 2026-07-22 Adverum Biotechnologies Inc COMPOSITIONS AND METHODS FOR THE TREATMENT OF ACHROMOTOPSY
CN116411024A (zh) * 2023-03-10 2023-07-11 上海泰昶生物技术有限公司 用于生产重组腺相关病毒的质粒系统
CN121046460A (zh) * 2024-05-31 2025-12-02 上海朗昇生物科技有限公司 用于治疗Prom1相关视网膜疾病的新型治疗药物

Family Cites Families (47)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3676007A (en) 1971-02-09 1972-07-11 Kiess Instr Inc Direct reading filter photometer
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
EP1007637B1 (en) 1997-04-14 2004-06-30 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant aav product
US6146874A (en) 1998-05-27 2000-11-14 University Of Florida Method of preparing recombinant adeno-associated virus compositions
EP1135468B1 (en) 1998-11-10 2010-01-06 University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
US6596535B1 (en) 1999-08-09 2003-07-22 Targeted Genetics Corporation Metabolically activated recombinant viral vectors and methods for the preparation and use
AU6814901A (en) 2000-06-01 2001-12-11 Univ North Carolina Methods and compounds for controlled release of recombinant parvovirus vectors
EP1409677A2 (en) 2001-01-19 2004-04-21 Incyte Genomics, Inc. Receptors and membrane-associated proteins
CA2442670A1 (en) 2001-04-13 2002-10-24 The Trustees Of The University Of Pennsylvania Method of treating or retarding the development of blindness
CN103589692B (zh) 2001-11-13 2018-07-24 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
EP1668143B1 (en) 2003-09-30 2013-03-20 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
DE23193606T1 (de) 2005-04-07 2024-02-15 The Trustees Of The University Of Pennsylvania Verfahren zur erhöhung der funktion eines aav-vektors
JP4495210B2 (ja) 2005-06-09 2010-06-30 パナソニック株式会社 振幅誤差補償装置及び直交度誤差補償装置
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
WO2010005533A2 (en) 2008-06-30 2010-01-14 The Johns Hopkins University Compositions and methods for the treatment of ocular oxidative stress and retinitis pigmentosa
WO2010099383A2 (en) 2009-02-26 2010-09-02 The Johns Hopkins University Compositions and methods for ex vivo hepatic nucleic acid delivery
US20120172419A1 (en) 2009-09-15 2012-07-05 Medical College Of Wisconsin Research Foundation Inc. Reagents and methods for modulating cone photoreceptor activity
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
CN103608455B (zh) 2011-01-07 2019-01-15 应用遗传科技公司 用于治疗色盲和其它疾病的启动子、表达盒、载体、药盒和方法
GB201103062D0 (en) 2011-02-22 2011-04-06 Isis Innovation Method
US9249425B2 (en) * 2011-05-16 2016-02-02 The Trustees Of The University Of Pennslyvania Proviral plasmids and production of recombinant adeno-associated virus
FR2977562B1 (fr) 2011-07-06 2016-12-23 Gaztransport Et Technigaz Cuve etanche et thermiquement isolante integree dans une structure porteuse
US9375491B2 (en) 2011-10-28 2016-06-28 University Of Florida Research Foundation, Inc. Chimeric promoter for cone photoreceptor targeted gene therapy
WO2013086515A1 (en) 2011-12-09 2013-06-13 The Johns Hopkins University Compositions and methods for the prevention or treatment of diabetic complications
WO2013151664A1 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins
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
JP6129180B2 (ja) 2012-08-08 2017-05-17 日本パーカライジング株式会社 金属表面処理液、金属基材の表面処理方法及びそれにより得られた金属基材
WO2014124282A1 (en) 2013-02-08 2014-08-14 The Trustees Of The University Of Pennsylvania Enhanced aav-mediated gene transfer for retinal therapies
DK2986635T3 (en) 2013-04-18 2019-01-28 Fond Telethon EFFECTIVE DELIVERY OF BIG GENES THROUGH DUAL-AAV VECTORS
EP2996725A4 (en) * 2013-05-16 2016-12-28 Applied Genetic Tech Corp PROMOTERS, EXPRESSION CASSETTES, VECTORS, KITS AND METHODS FOR TREATING COLOR BLINDNESS AND OTHER PROBLEMS
GB201322798D0 (en) 2013-12-20 2014-02-05 Oxford Biomedica Ltd Production system
ES2822551T3 (es) * 2014-03-17 2021-05-04 Adverum Biotechnologies Inc Composiciones y métodos para la expresión génica mejorada en células de los conos
EP3132051B1 (en) 2014-04-15 2019-03-20 Applied Genetic Technologies Corporation Codon optimized nucleic acid encoding a retinitis pigmentosa gtpase regulator (rpgr)
US20160206704A1 (en) 2015-01-16 2016-07-21 Isis Innovation Limited Method
CN109069668B (zh) * 2015-12-14 2023-04-18 宾夕法尼亚州大学信托人 用于眼病的基因疗法
US11827898B2 (en) * 2017-06-14 2023-11-28 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders

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