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
Prior art date
Application number
JP2020567085A
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English (en)
Japanese (ja)
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JP2021525531A (ja
JPWO2019232517A5 (https=
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
Publication of JPWO2019232517A5 publication Critical patent/JPWO2019232517A5/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 (4)

Publication Number Publication Date
JP2021525531A JP2021525531A (ja) 2021-09-27
JP2021525531A5 true JP2021525531A5 (https=) 2022-06-13
JPWO2019232517A5 JPWO2019232517A5 (https=) 2022-06-13
JP7563694B2 JP7563694B2 (ja) 2024-10-08

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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 (https=)
EP (1) EP3802836A4 (https=)
JP (1) JP7563694B2 (https=)
CN (2) CN112204145B (https=)
AU (1) AU2019276645B2 (https=)
CA (1) CA3102095A1 (https=)
IL (1) IL279091B1 (https=)
WO (1) WO2019232517A1 (https=)

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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
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
JP7334346B2 (ja) * 2020-07-24 2023-08-28 アールズィーノミクス・インコーポレイテッド ロドプシン転写体に特異的なトランススプライシングリボザイム及びその用途
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

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US8551970B2 (en) 1996-04-02 2013-10-08 Optigen Patents Limited Genetic suppression and replacement
US20040234999A1 (en) 1996-04-02 2004-11-25 Farrar Gwenyth Jane Genetic suppression and replacement
GB9606961D0 (en) 1996-04-02 1996-06-05 Farrar Gwyneth J Genetic strategy III
WO2004022722A2 (en) * 2002-09-06 2004-03-18 Massachusetts Institute Of Technology Lentiviral vectors, related reagents, and methods of use thereof
CA2522730A1 (en) 2003-04-18 2004-11-04 The Trustees Of The University Of Pennsylvania Compositions and methods for sirna inhibition of angiopoietin 1 and 2 and their receptor 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
EP1735442A2 (en) 2004-03-24 2006-12-27 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
US8257969B2 (en) 2007-04-12 2012-09-04 The Provost Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth Genetic suppression and replacement
US20110129825A1 (en) 2007-08-03 2011-06-02 Melba Stinnett 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
EP2855684A1 (en) 2012-05-25 2015-04-08 Commissariat à l'Énergie Atomique et aux Énergies Alternatives Vector for the selective silencing of a gene in astrocytes
WO2014138792A1 (en) 2013-03-14 2014-09-18 Commonwealth Scientific And Industrial Research Organisation Double-stranded rna
EP3071592B1 (en) 2013-11-20 2021-01-06 Fondazione Telethon Artificial dna-binding proteins and uses thereof
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BR112017017178A2 (pt) * 2015-02-26 2018-04-03 Ionis Pharmaceuticals Inc moduladores específicos de alelo de rodopsina de p23h
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EP3289080B1 (en) 2015-04-30 2021-08-25 The Trustees of Columbia University in the City of New York Gene therapy for autosomal dominant diseases
WO2017137493A1 (en) 2016-02-09 2017-08-17 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
CN109154002B (zh) 2016-03-01 2022-08-30 佛罗里达大学研究基金会有限公司 用于治疗显性视网膜色素变性的aav载体

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