JP2024119830A5 - - Google Patents

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Publication number
JP2024119830A5
JP2024119830A5 JP2024081593A JP2024081593A JP2024119830A5 JP 2024119830 A5 JP2024119830 A5 JP 2024119830A5 JP 2024081593 A JP2024081593 A JP 2024081593A JP 2024081593 A JP2024081593 A JP 2024081593A JP 2024119830 A5 JP2024119830 A5 JP 2024119830A5
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JP
Japan
Prior art keywords
seq
vector
shrna
nucleotide sequence
composition
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
JP2024081593A
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English (en)
Japanese (ja)
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JP2024119830A (ja
Filing date
Publication date
Priority claimed from JP2021511541A external-priority patent/JP2021534796A/ja
Application filed filed Critical
Publication of JP2024119830A publication Critical patent/JP2024119830A/ja
Publication of JP2024119830A5 publication Critical patent/JP2024119830A5/ja
Priority to JP2025266930A priority Critical patent/JP2026067864A/ja
Pending legal-status Critical Current

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JP2024081593A 2018-08-31 2024-05-20 ベスト病の処置のためのアデノ随伴ウイルスベクター Pending JP2024119830A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025266930A JP2026067864A (ja) 2018-08-31 2025-12-19 ベスト病の処置のためのアデノ随伴ウイルスベクター

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US201862726184P 2018-08-31 2018-08-31
US62/726,184 2018-08-31
US201862749622P 2018-10-23 2018-10-23
US62/749,622 2018-10-23
US201862754530P 2018-11-01 2018-11-01
US62/754,530 2018-11-01
JP2021511541A JP2021534796A (ja) 2018-08-31 2019-08-30 ベスト病の処置のためのアデノ随伴ウイルスベクター
PCT/US2019/049163 WO2020047476A1 (en) 2018-08-31 2019-08-30 Adeno-associated viral vectors for the treatment of best disease

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
JP2021511541A Division JP2021534796A (ja) 2018-08-31 2019-08-30 ベスト病の処置のためのアデノ随伴ウイルスベクター

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025266930A Division JP2026067864A (ja) 2018-08-31 2025-12-19 ベスト病の処置のためのアデノ随伴ウイルスベクター

Publications (2)

Publication Number Publication Date
JP2024119830A JP2024119830A (ja) 2024-09-03
JP2024119830A5 true JP2024119830A5 (enExample) 2024-12-23

Family

ID=69644668

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2021511541A Pending JP2021534796A (ja) 2018-08-31 2019-08-30 ベスト病の処置のためのアデノ随伴ウイルスベクター
JP2024081593A Pending JP2024119830A (ja) 2018-08-31 2024-05-20 ベスト病の処置のためのアデノ随伴ウイルスベクター
JP2025266930A Pending JP2026067864A (ja) 2018-08-31 2025-12-19 ベスト病の処置のためのアデノ随伴ウイルスベクター

Family Applications Before (1)

Application Number Title Priority Date Filing Date
JP2021511541A Pending JP2021534796A (ja) 2018-08-31 2019-08-30 ベスト病の処置のためのアデノ随伴ウイルスベクター

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025266930A Pending JP2026067864A (ja) 2018-08-31 2025-12-19 ベスト病の処置のためのアデノ随伴ウイルスベクター

Country Status (8)

Country Link
US (1) US20220033826A1 (enExample)
EP (1) EP3843791A4 (enExample)
JP (3) JP2021534796A (enExample)
CN (2) CN120350002A (enExample)
AU (2) AU2019328577A1 (enExample)
CA (1) CA3110563A1 (enExample)
IL (2) IL321380A (enExample)
WO (1) WO2020047476A1 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020047476A1 (en) * 2018-08-31 2020-03-05 University Of Florida Research Foundation, Incorporated Adeno-associated viral vectors for the treatment of best disease
US20220347318A1 (en) * 2019-09-25 2022-11-03 University Of Utah Research Foundation Methods and compositions for the expression of constitutively active rap1a from a vmd2 promoter
BR112022017182A2 (pt) * 2020-02-28 2022-11-08 Univ Pennsylvania Tratamento de bestrofinopatias autossômicas recessivas e métodos para avaliação das mesmas
WO2024064608A2 (en) * 2022-09-20 2024-03-28 Intergalactic Therapeutics, Inc. Best1 vectors and uses thereof

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050049212A1 (en) * 2001-10-09 2005-03-03 Arnd Steuernagel Bestrophin and bestrophin homologous proteins involved in the regulation of energy homeostasis
EP2305813A3 (en) * 2002-11-14 2012-03-28 Dharmacon, Inc. Fuctional and hyperfunctional sirna
WO2005056761A2 (en) * 2003-12-05 2005-06-23 Massachusetts Institute Of Technology REAGENTS AND METHODS FOR IDENTIFICATION OF RNAi PATHWAY GENES AND CHEMICAL MODULATORS OF RNAi
JP4543188B2 (ja) * 2004-03-10 2010-09-15 学校法人日本医科大学 ヒトチミジル酸合成酵素に対するRNAiとして作用するRNA配列
CN101538569B (zh) * 2008-08-19 2011-07-06 香港理工大学深圳研究院 抑制人dmt1蛋白的rna、重组体及应用
KR101154538B1 (ko) * 2009-08-24 2012-06-13 한국과학기술연구원 소뇌에서의 gaba 방출 조절제
CN102277434B (zh) * 2011-07-28 2015-11-18 上海吉凯基因化学技术有限公司 人zfx基因的用途及其相关药物
CN102533982B (zh) * 2011-12-19 2016-01-06 上海吉凯基因化学技术有限公司 人klf8基因在肿瘤治疗中的新用途
WO2013151664A1 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins
AU2013231839B2 (en) * 2012-03-16 2017-11-23 The Regents Of The University Of California A novel RNAi molecule delivery platform based on single-siRNA and shRNA nanocapsules
US9546366B2 (en) * 2013-03-14 2017-01-17 Raadysan Biotech, Inc. Replication factor C-40 (RFC40/RFC2) as a prognostic marker and target in estrogen positive and negative and triple negative breast cancer
EP3572513B1 (en) * 2013-12-09 2025-09-24 Baylor College of Medicine Hippo and dystrophin complex signaling in cardiomyocyte renewal
GB201322798D0 (en) * 2013-12-20 2014-02-05 Oxford Biomedica Ltd Production system
US20180346981A1 (en) * 2014-11-20 2018-12-06 Massachusetts Eye And Ear Infirmary Panel-based Genetic Diagnostic Testing for Inherited Eye Diseases
CA2972700A1 (en) * 2014-12-30 2016-07-07 Cell Cure Neurosciences Ltd. Methods of treating retinal diseases
CN107287238B (zh) * 2016-04-11 2020-10-16 厦门继景生物技术有限责任公司 一种基因载体及其用于治疗雷柏氏先天性黑矇2型疾病的基因治疗药物
BR112020020204A2 (pt) * 2018-04-05 2021-01-19 Oxford University Innovation Limited Composições e métodos para tratamento da distrofia macular
WO2020047476A1 (en) * 2018-08-31 2020-03-05 University Of Florida Research Foundation, Incorporated Adeno-associated viral vectors for the treatment of best disease

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