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
Application number
JP2021511541A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021534796A (ja
JPWO2020047476A5 (https=
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
Publication of JPWO2020047476A5 publication Critical patent/JPWO2020047476A5/ja
Priority to JP2024081593A priority Critical patent/JP2024119830A/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 (1)

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

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 (3)

Publication Number Publication Date
JP2021534796A JP2021534796A (ja) 2021-12-16
JP2021534796A5 true JP2021534796A5 (https=) 2022-09-07
JPWO2020047476A5 JPWO2020047476A5 (https=) 2022-09-07

Family

ID=69644668

Family Applications (2)

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

Family Applications After (1)

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

Country Status (8)

Country Link
US (1) US20220033826A1 (https=)
EP (1) EP3843791A4 (https=)
JP (2) JP2021534796A (https=)
CN (2) CN112839682B (https=)
AU (1) AU2019328577A1 (https=)
CA (1) CA3110563A1 (https=)
IL (2) IL321380A (https=)
WO (1) WO2020047476A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3110563A1 (en) * 2018-08-31 2020-03-05 University Of Florida Research Foundation, Incorporated Adeno-associated viral vectors for the treatment of best disease
KR20220106742A (ko) * 2019-09-25 2022-07-29 유니버시티 오브 유타 리서치 파운데이션 Vmd2 프로모터로부터 구성적으로 활성인 rap1a의 발현을 위한 방법 및 조성물
US20230112568A1 (en) * 2020-02-28 2023-04-13 The Trustees Of The University Of Pennsylvania Treating autosomal recessive bestrophinopathies and methods for evaluating same
EP4590841A2 (en) * 2022-09-20 2025-07-30 Aldevron, LLC Best1 vectors and uses thereof

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JP2005507398A (ja) * 2001-10-09 2005-03-17 デヴェロゲン アクチエンゲゼルシャフト フュア エントヴィックルングスビオローギッシェ フォルシュング エネルギー恒常性の調節に関与するBestrophinおよびBestrophin相同性タンパク質
EP2305812A3 (en) * 2002-11-14 2012-06-06 Dharmacon, Inc. Fuctional and hyperfunctional sirna
US20050266552A1 (en) * 2003-12-05 2005-12-01 Doench John G 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
JP6283652B2 (ja) * 2012-03-16 2018-02-21 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 単一のsiRNAおよびshRNAナノカプセルに基づいた新規RNAi分子送達プラットホーム
AU2013243948A1 (en) * 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. 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
EP4674963A3 (en) * 2013-12-09 2026-04-29 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
WO2016081885A2 (en) * 2014-11-20 2016-05-26 Massachusetts Eye And Ear Infirmary Panel-based genetic diagnostic testing for inherited eye diseases
CN114533761A (zh) * 2014-12-30 2022-05-27 细胞治疗神经科学有限公司 治疗视网膜疾病的方法
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
CA3110563A1 (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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