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
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JP2021506457A
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English (en)
Japanese (ja)
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JP2021532812A (ja
JP7615021B2 (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 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
Anticipated expiration legal-status Critical

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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 (3)

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

Family

ID=69413338

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

Family Applications After (1)

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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 (enExample)
EP (1) EP3833357A4 (enExample)
JP (2) JP7615021B2 (enExample)
KR (1) KR20210042123A (enExample)
CN (1) CN112805009A (enExample)
AU (1) AU2019317573A1 (enExample)
CA (1) CA3108293A1 (enExample)
IL (1) IL280636A (enExample)
WO (1) WO2020033473A1 (enExample)

Families Citing this family (22)

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Publication number Priority date Publication date Assignee Title
JP7013754B2 (ja) * 2017-09-15 2022-02-01 株式会社三洋物産 遊技機
JP7013753B2 (ja) * 2017-09-15 2022-02-01 株式会社三洋物産 遊技機
EP4013414A4 (en) * 2019-08-15 2023-09-27 The Children's Hospital of Philadelphia COMBINED INTRON-DERIVED MIRNA AND TRANSGENE THERAPY FOR THE TREATMENT OF SCA1
AU2020377204A1 (en) * 2019-11-01 2022-06-02 Novartis Ag The use of a splicing modulator for a treatment slowing progression of huntington's disease
CN115605596A (zh) * 2020-02-12 2023-01-13 费城儿童医院（Us） 用于基因表达的诱导型可变剪接调节的组合物和方法
JP2020151570A (ja) * 2020-06-24 2020-09-24 株式会社三洋物産 遊技機
US20240141384A1 (en) * 2021-02-19 2024-05-02 University Of Florida Research Foundation, Incoporated Methods and compositions to confer regulation to gene therapy cargoes by heterologous use of alternative splicing cassettes
JP2021087836A (ja) * 2021-03-01 2021-06-10 株式会社三洋物産 遊技機
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
JP2021087833A (ja) * 2021-03-01 2021-06-10 株式会社三洋物産 遊技機
EP4419692A4 (en) * 2021-10-22 2025-10-01 Fred Hutchinson Cancer Center SYNTHETIC INTRONS FOR TARGETED GENE EXPRESSION
US20250303000A1 (en) 2022-05-18 2025-10-02 The Children's Hospital Of Philadelphia Compositions and methods for inducible alternative splicing regulation of gene expression
CN119630687A (zh) * 2022-05-24 2025-03-14 凯特治疗学股份有限公司 用于治疗xlmtm的组合物
KR102470669B1 (ko) 2022-06-03 2022-11-25 충남대학교 산학협력단 스플라이싱-스위치 올리고뉴클레오티드를 유효성분으로 포함하는 폴리프로모 1 유전자의 스플라이싱 조절용 조성물
JP7556433B2 (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
JP7556432B2 (ja) * 2022-08-22 2024-09-26 株式会社三洋物産 遊技機
WO2024060205A1 (zh) * 2022-09-23 2024-03-28 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

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
DK2812004T3 (en) * 2012-02-10 2018-10-15 Ptc Therapeutics Inc COMPOUNDS FOR TREATMENT OF SPINAL MUSCLE DROPHY
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
EA201991309A1 (ru) * 2016-11-28 2019-11-29 Способы модуляции сплайсинга рнк
CN115605596A (zh) * 2020-02-12 2023-01-13 费城儿童医院（Us） 用于基因表达的诱导型可变剪接调节的组合物和方法

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