JP2021534796A5 - - Google Patents

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Publication number
JP2021534796A5
JP2021534796A5 JP2021511541A JP2021511541A JP2021534796A5 JP 2021534796 A5 JP2021534796 A5 JP 2021534796A5 JP 2021511541 A JP2021511541 A JP 2021511541A JP 2021511541 A JP2021511541 A JP 2021511541A JP 2021534796 A5 JP2021534796 A5 JP 2021534796A5
Authority
JP
Japan
Prior art keywords
nucleotide sequence
vector
shrna
seq
sequence
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
JP2021511541A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021534796A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/049163 external-priority patent/WO2020047476A1/en
Publication of JP2021534796A publication Critical patent/JP2021534796A/ja
Publication of JP2021534796A5 publication Critical patent/JP2021534796A5/ja
Priority to JP2024081593A priority Critical patent/JP2024119830A/ja
Priority to JP2025266930A priority patent/JP2026067864A/ja
Pending legal-status Critical Current

Links

JP2021511541A 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 Pending JP2021534796A (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2024081593A 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 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 (7)

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
PCT/US2019/049163 WO2020047476A1 (en) 2018-08-31 2019-08-30 Adeno-associated viral vectors for the treatment of best disease

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024081593A Division 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

Publications (2)

Publication Number Publication Date
JP2021534796A JP2021534796A (ja) 2021-12-16
JP2021534796A5 true JP2021534796A5 (enExample) 2022-09-07

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 After (2)

Application Number Title Priority Date Filing Date
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

Country Status (8)

Country Link
US (1) US20220033826A1 (enExample)
EP (1) EP3843791A4 (enExample)
JP (3) JP2021534796A (enExample)
CN (2) CN120350002A (enExample)
AU (1) 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
IL321380A (en) * 2018-08-31 2025-08-01 Univ Florida 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
IL295673A (en) * 2020-02-28 2022-10-01 Univ Pennsylvania Treatment of autosomal dominant strophin diseases and methods of their evaluation
WO2024064608A2 (en) * 2022-09-20 2024-03-28 Intergalactic Therapeutics, Inc. Best1 vectors and uses thereof

Family Cites Families (17)

* 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
AU2003295600A1 (en) * 2002-11-14 2004-06-15 Dharmacon, Inc. Functional 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
CA2907095C (en) * 2012-03-16 2020-11-03 The Regents Of The University Of California A novel rnai molecule delivery platform based on single-sirna and shrna nanocapsules
WO2013151665A2 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
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
JP6707028B2 (ja) * 2013-12-09 2020-06-10 ベイラー カレッジ オブ メディスンBaylor College Of Medicine 心筋細胞新生におけるhippo及びジストロフィン複合体シグナル伝達
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
EP3240612B1 (en) * 2014-12-30 2020-12-23 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
IL321380A (en) * 2018-08-31 2025-08-01 Univ Florida Adeno-associated viral vectors for the treatment of best disease

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