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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    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • 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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    • C12N15/09—Recombinant DNA-technology
    • 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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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination

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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 (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
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