JP2025016451A5 - - Google Patents

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Publication number
JP2025016451A5
JP2025016451A5 JP2024168632A JP2024168632A JP2025016451A5 JP 2025016451 A5 JP2025016451 A5 JP 2025016451A5 JP 2024168632 A JP2024168632 A JP 2024168632A JP 2024168632 A JP2024168632 A JP 2024168632A JP 2025016451 A5 JP2025016451 A5 JP 2025016451A5
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JP
Japan
Prior art keywords
vector
sequence
rna molecule
seq
synthetic rna
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2024168632A
Other languages
English (en)
Japanese (ja)
Other versions
JP2025016451A (ja
Filing date
Publication date
Priority claimed from JP2018545818A external-priority patent/JP7272795B2/ja
Application filed filed Critical
Publication of JP2025016451A publication Critical patent/JP2025016451A/ja
Publication of JP2025016451A5 publication Critical patent/JP2025016451A5/ja
Pending legal-status Critical Current

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JP2024168632A 2016-03-01 2024-09-27 優性網膜色素変性の治療のためのaavベクター Pending JP2025016451A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201662302122P 2016-03-01 2016-03-01
US62/302,122 2016-03-01
US201662398451P 2016-09-22 2016-09-22
US62/398,451 2016-09-22
JP2018545818A JP7272795B2 (ja) 2016-03-01 2017-03-01 優性網膜色素変性の治療のためのaavベクター
PCT/US2017/020289 WO2017151823A1 (en) 2016-03-01 2017-03-01 Aav vectors for treatment of dominant retinitis pigmentosa
JP2022156496A JP7642211B2 (ja) 2016-03-01 2022-09-29 優性網膜色素変性の治療のためのaavベクター

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
JP2022156496A Division JP7642211B2 (ja) 2016-03-01 2022-09-29 優性網膜色素変性の治療のためのaavベクター

Publications (2)

Publication Number Publication Date
JP2025016451A JP2025016451A (ja) 2025-02-04
JP2025016451A5 true JP2025016451A5 (enExample) 2025-05-30

Family

ID=59743213

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2018545818A Active JP7272795B2 (ja) 2016-03-01 2017-03-01 優性網膜色素変性の治療のためのaavベクター
JP2022156496A Active JP7642211B2 (ja) 2016-03-01 2022-09-29 優性網膜色素変性の治療のためのaavベクター
JP2024168632A Pending JP2025016451A (ja) 2016-03-01 2024-09-27 優性網膜色素変性の治療のためのaavベクター

Family Applications Before (2)

Application Number Title Priority Date Filing Date
JP2018545818A Active JP7272795B2 (ja) 2016-03-01 2017-03-01 優性網膜色素変性の治療のためのaavベクター
JP2022156496A Active JP7642211B2 (ja) 2016-03-01 2022-09-29 優性網膜色素変性の治療のためのaavベクター

Country Status (9)

Country Link
US (2) US11118185B2 (enExample)
EP (2) EP3423582B1 (enExample)
JP (3) JP7272795B2 (enExample)
CN (2) CN109154002B (enExample)
AU (2) AU2017227776C1 (enExample)
CA (1) CA3014671A1 (enExample)
ES (1) ES2923877T3 (enExample)
IL (2) IL261014B2 (enExample)
WO (1) WO2017151823A1 (enExample)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL261014B2 (en) 2016-03-01 2023-10-01 Univ Florida Aav vectors for treatment of dominant retinitis pigmentosa
EP3707257A4 (en) * 2017-11-07 2021-12-08 The University of North Carolina at Chapel Hill PROCEDURES AND COMPOSITIONS FOR CIRCULAR RNA MOLECULES
EP3793566A4 (en) * 2018-05-15 2022-03-16 University of Washington Compositions and methods for reducing spliceopathy and treating rna dominance disorders
JP7563694B2 (ja) * 2018-06-01 2024-10-08 ユニバーシティー オブ フロリダ リサーチ ファンデーション, インク. 優性網膜色素変性症の処置のための組成物および方法
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
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
CN113038971B (zh) * 2020-04-21 2022-06-24 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
CN111926044B (zh) * 2020-10-12 2021-01-22 北京大学第三医院(北京大学第三临床医学院) 结合突变rho基因的核酸分子和试剂盒
EP4284335A1 (en) 2021-02-01 2023-12-06 RegenxBio Inc. Gene therapy for neuronal ceroid lipofuscinoses
WO2025231487A1 (en) * 2024-05-03 2025-11-06 The Trustees Of The University Of Pennsylvania Photoreceptor specific promotors for use in gene therapies for treatment of retinal degeneration

Family Cites Families (25)

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Publication number Priority date Publication date Assignee Title
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
GB9606961D0 (en) 1996-04-02 1996-06-05 Farrar Gwyneth J Genetic strategy III
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
WO2003104413A2 (en) 2002-06-05 2003-12-18 University Of Florida Production of pseudotyped recombinant aav virions
WO2004094606A2 (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
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
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
EP2140004B1 (en) * 2007-04-12 2022-01-12 The Provost, Fellows, Foundation Scholars, & the other members of Board, of the College of the Holy & Undiv. Trinity of Queen Elizabeth near Dublin Genetic suppression and replacement
CA2702701A1 (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
CA2801523C (en) 2010-07-30 2021-08-03 Curevac Gmbh Complexation of nucleic acids with disulfide-crosslinked cationic components for transfection and immunostimulation
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
US20140289881A1 (en) 2013-03-14 2014-09-25 Commonwealth Scientific And Industrial Research Organisation Double-stranded rna
ES2760263T3 (es) 2014-03-21 2020-05-13 Genzyme Corp Tratamiento génico de la retinitis pigmentaria
BR112017017178A2 (pt) 2015-02-26 2018-04-03 Ionis Pharmaceuticals Inc moduladores específicos de alelo de rodopsina de p23h
EP4008780A1 (en) 2015-04-30 2022-06-08 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
IL261014B2 (en) 2016-03-01 2023-10-01 Univ Florida Aav vectors for treatment of dominant retinitis pigmentosa

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