JP2021532812A5 - - Google Patents

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Publication number
JP2021532812A5
JP2021532812A5 JP2021506457A JP2021506457A JP2021532812A5 JP 2021532812 A5 JP2021532812 A5 JP 2021532812A5 JP 2021506457 A JP2021506457 A JP 2021506457A JP 2021506457 A JP2021506457 A JP 2021506457A JP 2021532812 A5 JP2021532812 A5 JP 2021532812A5
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JP
Japan
Prior art keywords
pharmaceutical product
product according
protein
expression
rna
Prior art date
Application number
JP2021506457A
Other languages
English (en)
Japanese (ja)
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JPWO2020033473A5 (https=
JP7615021B2 (ja
JP2021532812A (ja
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Priority claimed from PCT/US2019/045401 external-priority patent/WO2020033473A1/en
Publication of JP2021532812A publication Critical patent/JP2021532812A/ja
Publication of JPWO2020033473A5 publication Critical patent/JPWO2020033473A5/ja
Publication of JP2021532812A5 publication Critical patent/JP2021532812A5/ja
Priority to JP2024231722A priority Critical patent/JP2025060942A/ja
Application granted granted Critical
Publication of JP7615021B2 publication Critical patent/JP7615021B2/ja
Active legal-status Critical Current
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JP2021506457A 2018-08-07 2019-08-07 遺伝子発現の選択的スプライシング調節および治療方法 Active JP7615021B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024231722A JP2025060942A (ja) 2018-08-07 2024-12-27 遺伝子発現の選択的スプライシング調節および治療方法

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201862715756P 2018-08-07 2018-08-07
US62/715,756 2018-08-07
US201962837701P 2019-04-23 2019-04-23
US62/837,701 2019-04-23
US201962838223P 2019-04-24 2019-04-24
US62/838,223 2019-04-24
US201962872417P 2019-07-10 2019-07-10
US62/872,417 2019-07-10
PCT/US2019/045401 WO2020033473A1 (en) 2018-08-07 2019-08-07 Alternative splicing regulation of gene expression and therapeutic methods

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024231722A Division JP2025060942A (ja) 2018-08-07 2024-12-27 遺伝子発現の選択的スプライシング調節および治療方法

Publications (4)

Publication Number Publication Date
JP2021532812A JP2021532812A (ja) 2021-12-02
JPWO2020033473A5 JPWO2020033473A5 (https=) 2022-07-26
JP2021532812A5 true JP2021532812A5 (https=) 2022-07-26
JP7615021B2 JP7615021B2 (ja) 2025-01-16

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Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021506457A Active JP7615021B2 (ja) 2018-08-07 2019-08-07 遺伝子発現の選択的スプライシング調節および治療方法
JP2024231722A Withdrawn JP2025060942A (ja) 2018-08-07 2024-12-27 遺伝子発現の選択的スプライシング調節および治療方法

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JP2024231722A Withdrawn JP2025060942A (ja) 2018-08-07 2024-12-27 遺伝子発現の選択的スプライシング調節および治療方法

Country Status (9)

Country Link
US (1) US20210355509A1 (https=)
EP (1) EP3833357A4 (https=)
JP (2) JP7615021B2 (https=)
KR (1) KR20210042123A (https=)
CN (1) CN112805009A (https=)
AU (1) AU2019317573A1 (https=)
CA (1) CA3108293A1 (https=)
IL (1) IL280636A (https=)
WO (1) WO2020033473A1 (https=)

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JP7013753B2 (ja) * 2017-09-15 2022-02-01 株式会社三洋物産 遊技機
JP7013754B2 (ja) * 2017-09-15 2022-02-01 株式会社三洋物産 遊技機
BR112022002794A2 (pt) * 2019-08-15 2022-08-09 Childrens Hospital Philadelphia Terapia combinada de transgene e mirna derivado de íntron para tratamento de sca1
TW202131920A (zh) * 2019-11-01 2021-09-01 瑞士商諾華公司 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途
EP4103717A4 (en) * 2020-02-12 2024-02-28 The Children's Hospital of Philadelphia COMPOSITIONS AND METHODS FOR INDUCIBLE ALTERNATIVE SPLICING REGULATION OF GENE EXPRESSION
JP2020151570A (ja) * 2020-06-24 2020-09-24 株式会社三洋物産 遊技機
EP4294459A4 (en) * 2021-02-19 2025-06-18 University Of Florida Research Foundation, Incorporated Methods and compositions to confer regulation to gene therapy cargoes by heterologous use of alternative splicing cassettes
JP2021079226A (ja) * 2021-03-01 2021-05-27 株式会社三洋物産 遊技機
JP2021079225A (ja) * 2021-03-01 2021-05-27 株式会社三洋物産 遊技機
JP2021087836A (ja) * 2021-03-01 2021-06-10 株式会社三洋物産 遊技機
JP2021087833A (ja) * 2021-03-01 2021-06-10 株式会社三洋物産 遊技機
US20250346893A1 (en) * 2021-10-22 2025-11-13 Fred Hutchinson Cancer Center Synthetic introns for targeted gene expression
EP4526455A1 (en) 2022-05-18 2025-03-26 The Children's Hospital of Philadelphia Compositions and methods for inducible alternative splicing regulation of gene expression
KR20250039324A (ko) * 2022-05-24 2025-03-20 케이트 테라퓨틱스, 인크. Xlmtm 치료를 위한 조성물
KR102470669B1 (ko) 2022-06-03 2022-11-25 충남대학교 산학협력단 스플라이싱-스위치 올리고뉴클레오티드를 유효성분으로 포함하는 폴리프로모 1 유전자의 스플라이싱 조절용 조성물
JP7556432B2 (ja) * 2022-08-22 2024-09-26 株式会社三洋物産 遊技機
JP7556434B2 (ja) * 2022-08-22 2024-09-26 株式会社三洋物産 遊技機
JP7556433B2 (ja) * 2022-08-22 2024-09-26 株式会社三洋物産 遊技機
CN118339290A (zh) * 2022-09-23 2024-07-12 北京基驭医疗科技有限公司 包含基于小分子药物的可变剪接调节元件的核酸分子
CN115747217B (zh) * 2022-10-26 2023-11-28 江苏省人民医院（南京医科大学第一附属医院） 长链非编码rna pdxdc1-as1及其应用
EP4701634A1 (en) * 2023-04-28 2026-03-04 Kriya Therapeutics, Inc. Riboswitches for regulating gene expression and therapeutic methods of using the same
WO2025170984A1 (en) * 2024-02-05 2025-08-14 The Children's Hospital Of Philadelphia Cell-type-specific control of gene expression in auditory hair cells

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CN1552849B (zh) * 1996-09-12 2013-08-14 辛根塔参与股份公司 表达纤维素分解酶的转基因植物
GB2404382B (en) * 2003-07-28 2008-01-30 Oxitec Ltd Pest control
US7838657B2 (en) * 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
GB2443186A (en) * 2006-10-25 2008-04-30 Oxitec Ltd Expression system for mediating alternative splicing
WO2009151546A2 (en) * 2008-05-27 2009-12-17 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
WO2010120820A1 (en) * 2009-04-13 2010-10-21 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing
EP2812004B1 (en) * 2012-02-10 2018-06-27 PTC Therapeutics, Inc. Compounds for treating spinal muscular atrophy
WO2014113540A1 (en) * 2013-01-16 2014-07-24 Iowa State University Research Foundation, Inc. A deep intronic target for splicing correction on spinal muscular atrophy gene
JP2019535789A (ja) * 2016-11-28 2019-12-12 ピーティーシー セラピューティクス，インコーポレーテッド Ｒｎａスプライシングを調節する方法
EP4103717A4 (en) * 2020-02-12 2024-02-28 The Children's Hospital of Philadelphia COMPOSITIONS AND METHODS FOR INDUCIBLE ALTERNATIVE SPLICING REGULATION OF GENE EXPRESSION

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