CN114127286A - 用于调节选择性内含子的剪接的方法和组合物 - Google Patents

用于调节选择性内含子的剪接的方法和组合物 Download PDF

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CN114127286A
CN114127286A CN202080040348.7A CN202080040348A CN114127286A CN 114127286 A CN114127286 A CN 114127286A CN 202080040348 A CN202080040348 A CN 202080040348A CN 114127286 A CN114127286 A CN 114127286A
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fold
mrna
aic
therapeutic agent
target protein
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伊莎贝尔·阿兹纳雷兹
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Stoke Therapeutics Inc
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Stoke Therapeutics Inc
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    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
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CN202080040348.7A 2019-04-26 2020-04-24 用于调节选择性内含子的剪接的方法和组合物 Pending CN114127286A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962839572P 2019-04-26 2019-04-26
US62/839,572 2019-04-26
PCT/US2020/029953 WO2020219977A1 (en) 2019-04-26 2020-04-24 Methods and compositions for modulating splicing of alternative introns

Publications (1)

Publication Number Publication Date
CN114127286A true CN114127286A (zh) 2022-03-01

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CN202080040348.7A Pending CN114127286A (zh) 2019-04-26 2020-04-24 用于调节选择性内含子的剪接的方法和组合物

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US (1) US20220162599A1 (https=)
EP (1) EP3959313A4 (https=)
JP (2) JP2022532998A (https=)
KR (1) KR20220019676A (https=)
CN (1) CN114127286A (https=)
AU (1) AU2020263581A1 (https=)
BR (1) BR112021021384A2 (https=)
CA (1) CA3134947A1 (https=)
MX (1) MX2021013081A (https=)
WO (1) WO2020219977A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025252001A1 (zh) * 2024-06-03 2025-12-11 时夕(广州)生物科技有限公司 调控RNA剪接的向导agRNA

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2023348346A1 (en) * 2022-09-30 2025-03-27 Contera Pharma A/S Antisense oligonucleotides for the treatment of canavan disease

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060058253A1 (en) * 2002-08-12 2006-03-16 Benoit Chabot Methods to reprogram splice site selection in pre-messenger rnas
WO2015108907A2 (en) * 2014-01-14 2015-07-23 Dana-Farber Cancer Institute, Inc. Compositions and methods for identification, assessment, prevention, and treatment of melanoma using pd-l1 isoforms
CN107109411A (zh) * 2014-10-03 2017-08-29 冷泉港实验室 核基因输出的定向增加
WO2019004939A1 (en) * 2017-06-27 2019-01-03 Agency For Science, Technology And Research ANTISENSE OLIGONUCLEOTIDES FOR MODULATING THE FUNCTION OF A LYMPHOCYTE T

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2555778A4 (en) * 2010-04-06 2014-05-21 Alnylam Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR INHIBITING CD274 / PD-L1 GENE EXPRESSION
WO2017100587A1 (en) * 2015-12-09 2017-06-15 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting programmed cell death 1 ligand 1 (pd-l1) and methods of use thereof
WO2017106377A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
RU2747822C2 (ru) * 2016-03-14 2021-05-14 Ф. Хоффманн-Ля Рош Аг Олигонуклеотиды для понижения экспрессии pd-l1

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060058253A1 (en) * 2002-08-12 2006-03-16 Benoit Chabot Methods to reprogram splice site selection in pre-messenger rnas
WO2015108907A2 (en) * 2014-01-14 2015-07-23 Dana-Farber Cancer Institute, Inc. Compositions and methods for identification, assessment, prevention, and treatment of melanoma using pd-l1 isoforms
CN107109411A (zh) * 2014-10-03 2017-08-29 冷泉港实验室 核基因输出的定向增加
WO2019004939A1 (en) * 2017-06-27 2019-01-03 Agency For Science, Technology And Research ANTISENSE OLIGONUCLEOTIDES FOR MODULATING THE FUNCTION OF A LYMPHOCYTE T

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
CHRISTOPHER R. SIBLEY 等: "Lessons from non-canonical splicing", 《NATURE REVIEWS GENETICS》, 31 May 2016 (2016-05-31), pages 3 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025252001A1 (zh) * 2024-06-03 2025-12-11 时夕(广州)生物科技有限公司 调控RNA剪接的向导agRNA

Also Published As

Publication number Publication date
EP3959313A4 (en) 2023-09-27
BR112021021384A2 (pt) 2021-12-21
US20220162599A1 (en) 2022-05-26
KR20220019676A (ko) 2022-02-17
AU2020263581A1 (en) 2021-12-16
MX2021013081A (es) 2021-12-10
WO2020219977A1 (en) 2020-10-29
EP3959313A1 (en) 2022-03-02
JP2022532998A (ja) 2022-07-21
JP2025128180A (ja) 2025-09-02
CA3134947A1 (en) 2020-10-29

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