CN112805009A - 基因表达的选择性剪接调控及治疗方法 - Google Patents

基因表达的选择性剪接调控及治疗方法 Download PDF

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CN112805009A
CN112805009A CN201980064769.0A CN201980064769A CN112805009A CN 112805009 A CN112805009 A CN 112805009A CN 201980064769 A CN201980064769 A CN 201980064769A CN 112805009 A CN112805009 A CN 112805009A
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protein
expression
aav
rna
pyridazin
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B·L·戴维森
A·M·莫内斯
A·A·亨德利
P·T·拉努姆
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Childrens Hospital of Philadelphia CHOP
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CN201980064769.0A 2018-08-07 2019-08-07 基因表达的选择性剪接调控及治疗方法 Pending CN112805009A (zh)

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

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CN112805009A true CN112805009A (zh) 2021-05-14

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CN201980064769.0A Pending CN112805009A (zh) 2018-08-07 2019-08-07 基因表达的选择性剪接调控及治疗方法

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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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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
CN115747217A (zh) * 2022-10-26 2023-03-07 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

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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
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
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