JP2021520837A5 - - Google Patents

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Publication number
JP2021520837A5
JP2021520837A5 JP2020557945A JP2020557945A JP2021520837A5 JP 2021520837 A5 JP2021520837 A5 JP 2021520837A5 JP 2020557945 A JP2020557945 A JP 2020557945A JP 2020557945 A JP2020557945 A JP 2020557945A JP 2021520837 A5 JP2021520837 A5 JP 2021520837A5
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JP
Japan
Prior art keywords
nucleic acid
nucleotides
abca4
exons
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.)
Granted
Application number
JP2020557945A
Other languages
English (en)
Japanese (ja)
Other versions
JP7561624B2 (ja
JP2021520837A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/027981 external-priority patent/WO2019204514A1/en
Publication of JP2021520837A publication Critical patent/JP2021520837A/ja
Publication of JP2021520837A5 publication Critical patent/JP2021520837A5/ja
Priority to JP2024073380A priority Critical patent/JP2024099770A/ja
Application granted granted Critical
Publication of JP7561624B2 publication Critical patent/JP7561624B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2020557945A 2018-04-17 2019-04-17 トランススプライシング分子 Active JP7561624B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024073380A JP2024099770A (ja) 2018-04-17 2024-04-30 トランススプライシング分子

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862658667P 2018-04-17 2018-04-17
US201862658658P 2018-04-17 2018-04-17
US62/658,658 2018-04-17
US62/658,667 2018-04-17
PCT/US2019/027981 WO2019204514A1 (en) 2018-04-17 2019-04-17 Trans-splicing molecules

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024073380A Division JP2024099770A (ja) 2018-04-17 2024-04-30 トランススプライシング分子

Publications (3)

Publication Number Publication Date
JP2021520837A JP2021520837A (ja) 2021-08-26
JP2021520837A5 true JP2021520837A5 (enExample) 2022-04-25
JP7561624B2 JP7561624B2 (ja) 2024-10-04

Family

ID=68239871

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020557945A Active JP7561624B2 (ja) 2018-04-17 2019-04-17 トランススプライシング分子
JP2024073380A Pending JP2024099770A (ja) 2018-04-17 2024-04-30 トランススプライシング分子

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024073380A Pending JP2024099770A (ja) 2018-04-17 2024-04-30 トランススプライシング分子

Country Status (11)

Country Link
US (3) US11993776B2 (enExample)
EP (1) EP3781213A4 (enExample)
JP (2) JP7561624B2 (enExample)
KR (2) KR102866133B1 (enExample)
CN (1) CN112449605A (enExample)
AU (1) AU2019255708B2 (enExample)
BR (1) BR112020021228A2 (enExample)
CA (1) CA3097004A1 (enExample)
IL (1) IL278019A (enExample)
MX (2) MX2020010959A (enExample)
WO (1) WO2019204514A1 (enExample)

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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
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
AU2022337146A1 (en) 2021-09-03 2024-03-14 Tacit Therapeutics, Inc. Rna editing via recruitment of spliceosome components
EP4522758A4 (en) * 2022-05-13 2026-05-06 Ascidian Therapeutics Inc Abca4 trans-splicing molecules
US20260007773A1 (en) * 2022-07-07 2026-01-08 Aldevron, Llc Ocular vectors and uses thereof
EP4594505A1 (en) * 2022-09-30 2025-08-06 Centre National de la Recherche Scientifique Therapy by trans-splicing of opa1 pre-messenger rnas for the treatment of diseases associated with opa1 gene mutations
KR20250157548A (ko) * 2023-02-15 2025-11-04 아시디엔 테라퓨틱스, 인크. Htt 트랜스 스플라이싱 분자
WO2025166360A1 (en) * 2024-02-03 2025-08-07 Duke University Compositions for and methods of engineering the transcriptome
WO2026012439A1 (en) * 2024-07-11 2026-01-15 Sichuan Real & Best Biotech Co., Ltd. Rna trans-splicing molecules and systems

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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
WO2020214990A1 (en) 2019-04-17 2020-10-22 Ascidian Therapeutics, Inc. Abca4 cell line and methods of use
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EP4522758A4 (en) 2022-05-13 2026-05-06 Ascidian Therapeutics Inc Abca4 trans-splicing molecules

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