CA3108293A1 - Alternative splicing regulation of gene expression and therapeutic methods - Google Patents

Alternative splicing regulation of gene expression and therapeutic methods Download PDF

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Publication number
CA3108293A1
CA3108293A1 CA3108293A CA3108293A CA3108293A1 CA 3108293 A1 CA3108293 A1 CA 3108293A1 CA 3108293 A CA3108293 A CA 3108293A CA 3108293 A CA3108293 A CA 3108293A CA 3108293 A1 CA3108293 A1 CA 3108293A1
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Prior art keywords
protein
expression
aav
rna
disease
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CA3108293A
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English (en)
French (fr)
Inventor
Beverly L. Davidson
Alejandro Mas Monteys
Amiel Al HUNDLEY
Paul T. RANUM
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Childrens Hospital of Philadelphia CHOP
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Childrens Hospital of Philadelphia CHOP
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    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
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    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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CA3108293A 2018-08-07 2019-08-07 Alternative splicing regulation of gene expression and therapeutic methods Pending CA3108293A1 (en)

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

Publications (1)

Publication Number Publication Date
CA3108293A1 true CA3108293A1 (en) 2020-02-13

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Application Number Title Priority Date Filing Date
CA3108293A Pending CA3108293A1 (en) 2018-08-07 2019-08-07 Alternative splicing regulation of gene expression and therapeutic methods

Country Status (9)

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

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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
JP2022545378A (ja) * 2019-08-15 2022-10-27 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア ＳＣＡ１の治療のための、導入遺伝子およびイントロン由来ｍｉＲＮＡを組み合わせた治療法
EP4051280A1 (en) * 2019-11-01 2022-09-07 Novartis AG The use of a splicing modulator for a treatment slowing progression of huntington's disease
US20230340498A1 (en) * 2020-02-12 2023-10-26 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
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
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
EP4419692A4 (en) * 2021-10-22 2025-10-01 Fred Hutchinson Cancer Center SYNTHETIC INTRONS FOR TARGETED GENE EXPRESSION
JP2025521120A (ja) 2022-05-18 2025-07-08 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア 遺伝子発現の誘導性選択的スプライシング調節のための組成物および方法
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
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
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
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