JP2021525531A5 - - Google Patents

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Publication number
JP2021525531A5
JP2021525531A5 JP2020567085A JP2020567085A JP2021525531A5 JP 2021525531 A5 JP2021525531 A5 JP 2021525531A5 JP 2020567085 A JP2020567085 A JP 2020567085A JP 2020567085 A JP2020567085 A JP 2020567085A JP 2021525531 A5 JP2021525531 A5 JP 2021525531A5
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JP
Japan
Prior art keywords
vector
seq
nucleotide sequence
raav
shrna
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JP2020567085A
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English (en)
Japanese (ja)
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JP2021525531A (ja
JP7563694B2 (ja
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Priority claimed from PCT/US2019/035159 external-priority patent/WO2019232517A1/en
Publication of JP2021525531A publication Critical patent/JP2021525531A/ja
Publication of JP2021525531A5 publication Critical patent/JP2021525531A5/ja
Application granted granted Critical
Publication of JP7563694B2 publication Critical patent/JP7563694B2/ja
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JP2020567085A 2018-06-01 2019-06-03 優性網膜色素変性症の処置のための組成物および方法 Active JP7563694B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862679585P 2018-06-01 2018-06-01
US62/679,585 2018-06-01
US201962809539P 2019-02-22 2019-02-22
US62/809,539 2019-02-22
PCT/US2019/035159 WO2019232517A1 (en) 2018-06-01 2019-06-03 Compositions and methods for treatment of dominant retinitis pigmentosa

Publications (3)

Publication Number Publication Date
JP2021525531A JP2021525531A (ja) 2021-09-27
JP2021525531A5 true JP2021525531A5 (enExample) 2022-06-13
JP7563694B2 JP7563694B2 (ja) 2024-10-08

Family

ID=68698456

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2020567085A Active JP7563694B2 (ja) 2018-06-01 2019-06-03 優性網膜色素変性症の処置のための組成物および方法

Country Status (8)

Country Link
US (2) US12203074B2 (enExample)
EP (1) EP3802836A4 (enExample)
JP (1) JP7563694B2 (enExample)
CN (2) CN112204145B (enExample)
AU (1) AU2019276645B2 (enExample)
CA (1) CA3102095A1 (enExample)
IL (1) IL279091B2 (enExample)
WO (1) WO2019232517A1 (enExample)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021212686A1 (zh) * 2020-04-21 2021-10-28 北京中因科技有限公司 RHO-adRP基于基因编辑的方法和组合物
WO2022019706A1 (ko) * 2020-07-24 2022-01-27 알지노믹스 주식회사 로돕신 전사체에 특이적인 트랜스-스플라이싱 라이보자임 및 이의 용도
CN114521214A (zh) * 2020-07-24 2022-05-20 尔知渃米斯股份有限公司 视紫红质转录体特异性反式剪接核酶及其用途
US20220098615A1 (en) * 2020-09-30 2022-03-31 NGGT, Inc. Dual functional expression vectors and methods of use thereof
WO2024211266A1 (en) * 2023-04-07 2024-10-10 University Of Houston System Downregulation of rhodopsin as a therapeutic strategy for peripherin-2-associated inherited retinal disorders

Family Cites Families (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB9606961D0 (en) 1996-04-02 1996-06-05 Farrar Gwyneth J Genetic strategy III
US20040234999A1 (en) 1996-04-02 2004-11-25 Farrar Gwenyth Jane Genetic suppression and replacement
US8551970B2 (en) 1996-04-02 2013-10-08 Optigen Patents Limited Genetic suppression and replacement
WO2004022722A2 (en) * 2002-09-06 2004-03-18 Massachusetts Institute Of Technology Lentiviral vectors, related reagents, and methods of use thereof
EP1615943A4 (en) 2003-04-18 2006-08-16 Univ Pennsylvania COMPOSITION AND METHOD FOR SIRNA INHIBITION OF ANGIOPOIETIN 1 AND 2 AND THEIR RECIPIENTS TIE2
US20070009899A1 (en) 2003-10-02 2007-01-11 Mounts William M Nucleic acid arrays for detecting gene expression in animal models of inflammatory diseases
WO2005090572A2 (en) 2004-03-24 2005-09-29 Oncotherapy Science, Inc. Compositions and methods for treating pancreatic cancer
US20080221057A1 (en) 2007-02-16 2008-09-11 Wyeth Secreted protein ccdc80 regulates adipocyte differentiation
CA2683469C (en) 2007-04-12 2019-11-26 The Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth Genetic suppression and replacement
WO2009035792A1 (en) 2007-08-03 2009-03-19 Melba Ketchum Compositions, methods and systems for the simultaneous determination of parentage, identity, sex, genotype and/or phenotype and breed determination in animals
US8450473B2 (en) * 2009-04-30 2013-05-28 The Research Foundation Of State University Of New York Compositions and methods for therapy of macular degeneration
CN102061308B (zh) 2010-10-29 2013-05-08 北京未名凯拓作物设计中心有限公司 一种用百草枯筛选转基因植物的方法
CN102061305B (zh) 2010-12-15 2012-07-18 深圳市百恩维生物科技有限公司 一种多ShRNA高效协同沉默基因方法及载体
US20130064815A1 (en) 2011-09-12 2013-03-14 The Trustees Of Princeton University Inducing apoptosis in quiescent cells
US9695443B2 (en) 2012-05-25 2017-07-04 Commissariat A L'energie Atomique Et Aux Energies Alternatives Vector for the selective silencing of a gene in astrocytes
US20140289881A1 (en) 2013-03-14 2014-09-25 Commonwealth Scientific And Industrial Research Organisation Double-stranded rna
WO2015075154A2 (en) 2013-11-20 2015-05-28 Fondazione Telethon Artificial dna-binding proteins and uses thereof
DK3628334T5 (da) 2014-03-21 2024-09-02 Genzyme Corp Genterapi til behandling af retinitis pigmentosa
EP3137497B2 (en) * 2014-05-02 2025-03-05 Genzyme Corporation Aav vectors for retinal and cns gene therapy
US10426789B2 (en) * 2015-02-26 2019-10-01 Ionis Pharmaceuticals, Inc. Allele specific modulators of P23H rhodopsin
PT3265568T (pt) 2015-03-06 2020-08-20 Massachusetts Eye & Ear Infirmary Terapias de aumento de genes para a degeneração hereditária da retina causada por mutações no gene prpf31
US20190275168A1 (en) 2015-04-30 2019-09-12 The Trustees Of Columbia University In The City Of New York Gene therapy for autosomal dominant diseases
US11096955B2 (en) 2016-02-09 2021-08-24 Fondazione Telethon Synthetic promoters and uses thereof
US20170348387A1 (en) * 2016-02-29 2017-12-07 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for nphp5 lca-ciliopathy
US11118185B2 (en) 2016-03-01 2021-09-14 University Of Florida Research Foundation, Incorporated AAV vectors for treatment of dominant retinitis pigmentosa

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