CN114040974B - 用于高效rna反式剪接的三螺旋终止子 - Google Patents

用于高效rna反式剪接的三螺旋终止子

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Publication number
CN114040974B
CN114040974B CN202080044745.1A CN202080044745A CN114040974B CN 114040974 B CN114040974 B CN 114040974B CN 202080044745 A CN202080044745 A CN 202080044745A CN 114040974 B CN114040974 B CN 114040974B
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sequence
domain
trans
gene
splicing
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CN114040974A (zh
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K·J·费希尔
J·班尼特
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University of Pennsylvania Penn
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University of Pennsylvania Penn
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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N15/86—Viral vectors
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    • C12N2320/33—Alteration of splicing
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
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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
    • C—CHEMISTRY; METALLURGY
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
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  • Pharmacology & Pharmacy (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
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  • Biochemistry (AREA)
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  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN202080044745.1A 2019-04-17 2020-04-17 用于高效rna反式剪接的三螺旋终止子 Active CN114040974B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962835164P 2019-04-17 2019-04-17
US62/835164 2019-04-17
PCT/US2020/028797 WO2020214973A1 (en) 2019-04-17 2020-04-17 Triple helix terminator for efficient rna trans-splicing

Publications (2)

Publication Number Publication Date
CN114040974A CN114040974A (zh) 2022-02-11
CN114040974B true CN114040974B (zh) 2025-11-04

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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)

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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

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
A triple helix stabilizes the 39 ends of long noncoding RNAs that lack poly(A) tails;WILUSZ J.E.等;《GENES & DEVELOPMENT》;第26卷(第21期);第2392-2407页 *
Spliceosome-Mediated Pre-mRNA trans-Splicing Can Repair CEP290 mRNA;DOOLEY S.J.等;《Nucleic Acids》;第12卷;第294-308页 *

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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
CN114040974A (zh) 2022-02-11
EP3956442A4 (en) 2023-01-25
IL287243A (en) 2021-12-01
BR112021020539A2 (pt) 2022-01-04
KR20220002910A (ko) 2022-01-07
EP3956442A1 (en) 2022-02-23

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