JP2024518817A5 - - Google Patents

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Publication number
JP2024518817A5
JP2024518817A5 JP2023563303A JP2023563303A JP2024518817A5 JP 2024518817 A5 JP2024518817 A5 JP 2024518817A5 JP 2023563303 A JP2023563303 A JP 2023563303A JP 2023563303 A JP2023563303 A JP 2023563303A JP 2024518817 A5 JP2024518817 A5 JP 2024518817A5
Authority
JP
Japan
Prior art keywords
trans
guanine
uracil
group
splicing
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2023563303A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024518817A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2022/020151 external-priority patent/WO2022220968A1/en
Publication of JP2024518817A publication Critical patent/JP2024518817A/ja
Publication of JP2024518817A5 publication Critical patent/JP2024518817A5/ja
Pending legal-status Critical Current

Links

JP2023563303A 2021-04-15 2022-03-14 ヒト細胞における標的ｒｎａ配列の置換のための高効率トランススプライシング Pending JP2024518817A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163175223P 2021-04-15 2021-04-15
US63/175,223 2021-04-15
PCT/US2022/020151 WO2022220968A1 (en) 2021-04-15 2022-03-14 High efficiency trans-splicing for replacement of targeted rna sequences in human cells

Publications (2)

Publication Number Publication Date
JP2024518817A JP2024518817A (ja) 2024-05-07
JP2024518817A5 true JP2024518817A5 (https=) 2025-03-21

Family

ID=83640536

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023563303A Pending JP2024518817A (ja) 2021-04-15 2022-03-14 ヒト細胞における標的ｒｎａ配列の置換のための高効率トランススプライシング

Country Status (6)

Country Link
US (1) US20240209366A1 (https=)
EP (1) EP4323391A4 (https=)
JP (1) JP2024518817A (https=)
CN (1) CN117715927A (https=)
AU (1) AU2022259416A1 (https=)
WO (1) WO2022220968A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4522758A4 (en) 2022-05-13 2026-05-06 Ascidian Therapeutics Inc Abca4 trans-splicing molecules
WO2025147110A1 (ko) * 2024-01-02 2025-07-10 알지노믹스 주식회사 Mecp2 전사체 특이적 트랜스-스플라이싱 리보자임 및 이의 용도
WO2026012439A1 (en) * 2024-07-11 2026-01-15 Sichuan Real & Best Biotech Co., Ltd. Rna trans-splicing molecules and systems
CN120082561A (zh) * 2025-03-06 2025-06-03 西安艾领克生物科技有限公司 一种重组rna适配体及其制备方法和应用

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004525618A (ja) * 2001-01-08 2004-08-26 イントロン，インコーポレーテッド スプライセオソームにより媒介されるｒｎａトランススプライシング
DE10139492B4 (de) * 2001-08-13 2004-06-09 Eul, Joachim, Dr. Verfahren zur Reparatur einer mutierten RNA aus einer gendefekten DNA und zum gezielten Abtöten von Tumorzellen durch RNA-Transspleißen sowie Verfahren zum Nachweis von natürlich-transgespleißter zellulärer RNA
EP1521766B1 (en) * 2002-06-05 2012-11-07 VIRxSYS Corporation SPLICEOSOME MEDIATED RNA i TRANS /i -SPLICING AND CORRECTION OF FACTOR VIII GENETIC DEFECTS USING SPLICEOSOME MEDIATED RNA TRANS SPLING
EP2151248A1 (en) * 2008-07-30 2010-02-10 Johann Bauer Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses
US9303078B2 (en) * 2009-10-08 2016-04-05 Association Institut De Myologie Nucleic acid molecules and methods for exchanging exon(s) by transsplicing
EP3436590A4 (en) * 2016-04-01 2019-12-04 National University of Singapore TRANSSPLEISSEN OF RNA (TSRNA)
EP3510161A4 (en) * 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON
SG11202106356QA (en) * 2018-12-20 2021-07-29 Vigeneron Gmbh An optimized acceptor splice site module for biological and biotechnological applications
WO2020205604A1 (en) * 2019-03-29 2020-10-08 Salk Institute For Biological Studies High-efficiency reconstitution of rna molecules
BR112021020539A2 (pt) * 2019-04-17 2022-01-04 Univ Pennsylvania Terminador de hélice tripla para trans-splicing de rna eficiente
CN115011598A (zh) * 2020-09-02 2022-09-06 西湖大学 杜氏肌营养不良症相关的外显子剪接增强子、sgRNA、基因编辑工具及应用
EP4217010A4 (en) * 2020-09-28 2025-01-08 Tacit Therapeutics, Inc. TRANS-SPLICING SYSTEM FOR TISSUE-SPECIFIC REPLACEMENT OF RNA SEQUENCES
AU2022337146A1 (en) * 2021-09-03 2024-03-14 Tacit Therapeutics, Inc. Rna editing via recruitment of spliceosome components

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