JP2024518817A - 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 - Google Patents

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 Download PDF

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JP2024518817A
JP2024518817A JP2023563303A JP2023563303A JP2024518817A JP 2024518817 A JP2024518817 A JP 2024518817A JP 2023563303 A JP2023563303 A JP 2023563303A JP 2023563303 A JP2023563303 A JP 2023563303A JP 2024518817 A JP2024518817 A JP 2024518817A
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splicing
trans
rna
nucleotides
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ネルズ，デヴィッド・アレン
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タシト・セラピューティクス・インコーポレーテッド
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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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)

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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

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

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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)

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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

Citations (9)

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JP2005528911A (ja) * 2002-06-05 2005-09-29 イントロン，インコーポレーテッド スプライセオソーム媒介型ｒｎａトランススプライシング、及びスプライセオソーム媒介型ｒｎａトランススプライシングを利用した第ｖｉｉｉ因子の遺伝的欠陥の修正
WO2011042556A1 (en) * 2009-10-08 2011-04-14 Association Institut De Myologie Nucleic acid molecules and methods for exchanging exon(s) by transsplicing
JP2011529333A (ja) * 2008-07-30 2011-12-08 ヨハン バウアー 改善されたプレ−ｍＲＮＡトランススプライシング分子（ＲＴＭ）分子およびその使用
JP2019509756A (ja) * 2016-04-01 2019-04-11 ナショナル ユニバーシティ オブ シンガポール トランス−スプライシングＲＮＡ（ｔｓＲＮＡ）
WO2020205604A1 (en) * 2019-03-29 2020-10-08 Salk Institute For Biological Studies High-efficiency reconstitution of rna molecules
WO2020214973A1 (en) * 2019-04-17 2020-10-22 The Trustees Of The University Of Pennsylvania Triple helix terminator for efficient rna trans-splicing
CN112063621A (zh) * 2020-09-02 2020-12-11 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
WO2022067228A1 (en) * 2020-09-28 2022-03-31 U1 Bio, Inc. Trans-splicing system for tissue-specific replacement of rna sequences
JP2024533163A (ja) * 2021-09-03 2024-09-12 タシト・セラピューティクス・インコーポレーテッド スプライソソーム構成要素のリクルートを介したｒｎａ編集

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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
EP3510161A4 (en) * 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON
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JP2005528911A (ja) * 2002-06-05 2005-09-29 イントロン，インコーポレーテッド スプライセオソーム媒介型ｒｎａトランススプライシング、及びスプライセオソーム媒介型ｒｎａトランススプライシングを利用した第ｖｉｉｉ因子の遺伝的欠陥の修正
JP2011529333A (ja) * 2008-07-30 2011-12-08 ヨハン バウアー 改善されたプレ−ｍＲＮＡトランススプライシング分子（ＲＴＭ）分子およびその使用
WO2011042556A1 (en) * 2009-10-08 2011-04-14 Association Institut De Myologie Nucleic acid molecules and methods for exchanging exon(s) by transsplicing
JP2019509756A (ja) * 2016-04-01 2019-04-11 ナショナル ユニバーシティ オブ シンガポール トランス−スプライシングＲＮＡ（ｔｓＲＮＡ）
WO2020205604A1 (en) * 2019-03-29 2020-10-08 Salk Institute For Biological Studies High-efficiency reconstitution of rna molecules
WO2020214973A1 (en) * 2019-04-17 2020-10-22 The Trustees Of The University Of Pennsylvania Triple helix terminator for efficient rna trans-splicing
CN112063621A (zh) * 2020-09-02 2020-12-11 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
WO2022067228A1 (en) * 2020-09-28 2022-03-31 U1 Bio, Inc. Trans-splicing system for tissue-specific replacement of rna sequences
JP2024533163A (ja) * 2021-09-03 2024-09-12 タシト・セラピューティクス・インコーポレーテッド スプライソソーム構成要素のリクルートを介したｒｎａ編集

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WO2022220968A1 (en) 2022-10-20
EP4323391A4 (en) 2025-04-16
EP4323391A1 (en) 2024-02-21
US20240209366A1 (en) 2024-06-27
CN117715927A (zh) 2024-03-15
AU2022259416A1 (en) 2023-11-16

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