KR20220002910A - 효율적인 rna 트랜스-스플라이싱을 위한 삼중 나선 종결인자 - Google Patents

효율적인 rna 트랜스-스플라이싱을 위한 삼중 나선 종결인자 Download PDF

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KR20220002910A
KR20220002910A KR1020217034092A KR20217034092A KR20220002910A KR 20220002910 A KR20220002910 A KR 20220002910A KR 1020217034092 A KR1020217034092 A KR 1020217034092A KR 20217034092 A KR20217034092 A KR 20217034092A KR 20220002910 A KR20220002910 A KR 20220002910A
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splicing
domain
nucleic acid
trans
sequence
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크리쉬나 제이. 피셔
진 베넷
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더 트러스티스 오브 더 유니버시티 오브 펜실베니아
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KR1020217034092A 2019-04-17 2020-04-17 효율적인 rna 트랜스-스플라이싱을 위한 삼중 나선 종결인자 Pending KR20220002910A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962835164P 2019-04-17 2019-04-17
US62/835,164 2019-04-17
PCT/US2020/028797 WO2020214973A1 (en) 2019-04-17 2020-04-17 Triple helix terminator for efficient rna trans-splicing

Publications (1)

Publication Number Publication Date
KR20220002910A true KR20220002910A (ko) 2022-01-07

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KR1020217034092A Pending KR20220002910A (ko) 2019-04-17 2020-04-17 효율적인 rna 트랜스-스플라이싱을 위한 삼중 나선 종결인자

Country Status (11)

Country Link
US (1) US20220204989A1 (https=)
EP (1) EP3956442A4 (https=)
JP (2) JP2022529065A (https=)
KR (1) KR20220002910A (https=)
CN (1) CN114040974B (https=)
AU (1) AU2020260154A1 (https=)
BR (1) BR112021020539A2 (https=)
CA (1) CA3133555A1 (https=)
IL (1) IL287243A (https=)
MX (1) MX2021012702A (https=)
WO (1) WO2020214973A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102866133B1 (ko) 2018-04-17 2025-09-30 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 트랜스-스플라이싱 분자
BR112021020539A2 (pt) * 2019-04-17 2022-01-04 Univ Pennsylvania Terminador de hélice tripla para trans-splicing de rna eficiente
WO2022220968A1 (en) * 2021-04-15 2022-10-20 U1 Bio, Inc. High efficiency trans-splicing for replacement of targeted rna sequences in human cells
AU2022337146A1 (en) 2021-09-03 2024-03-14 Tacit Therapeutics, Inc. Rna editing via recruitment of spliceosome components
EP4511488A2 (en) * 2022-04-20 2025-02-26 Tacit Therapeutics, Inc. Stabilization of therapeutic trans-splicing rna molecules in human cells
WO2023215761A1 (en) * 2022-05-03 2023-11-09 Tacit Therapeutics, Inc. Localization of trans-splicing nucleic acid molecules to and within the cellular nucleus
EP4522758A4 (en) * 2022-05-13 2026-05-06 Ascidian Therapeutics Inc Abca4 trans-splicing molecules
WO2024112957A1 (en) 2022-11-23 2024-05-30 Amber Bio Inc. Gene-modifying endonucleases

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2151248A1 (en) * 2008-07-30 2010-02-10 Johann Bauer Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses
US9717749B2 (en) * 2012-10-16 2017-08-01 Massachusetts Institute Of Technology Production of stable non-polyadenylated RNAs
GB201219762D0 (en) * 2012-11-02 2012-12-19 Bauer Johann A RNA trans-splicing molecule (RTM) for use in the treatment of cancer
AU2014255665B2 (en) * 2013-04-18 2018-08-02 Fondazione Telethon Effective delivery of large genes by dual AAV vectors
CA3005474A1 (en) * 2015-11-19 2017-05-26 The Trustees Of The University Of Pennsylvania Compositions and methods for correction of heritable ocular disease
WO2019027869A1 (en) * 2017-07-31 2019-02-07 Massachusetts Institute Of Technology RNA-CLUSTER INDUCED TRANSCRIPT STABILIZER AND USES THEREOF
KR102866133B1 (ko) * 2018-04-17 2025-09-30 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 트랜스-스플라이싱 분자
BR112021020539A2 (pt) * 2019-04-17 2022-01-04 Univ Pennsylvania Terminador de hélice tripla para trans-splicing de rna eficiente

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Publication number Publication date
US20220204989A1 (en) 2022-06-30
WO2020214973A1 (en) 2020-10-22
CA3133555A1 (en) 2020-10-22
JP2025121988A (ja) 2025-08-20
MX2021012702A (es) 2022-01-24
AU2020260154A1 (en) 2021-11-11
JP2022529065A (ja) 2022-06-16
CN114040974B (zh) 2025-11-04
CN114040974A (zh) 2022-02-11
EP3956442A4 (en) 2023-01-25
IL287243A (en) 2021-12-01
BR112021020539A2 (pt) 2022-01-04
EP3956442A1 (en) 2022-02-23

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