CN115023499A - 使用CRISPR/Cas13的靶向反式剪接 - Google Patents

使用CRISPR/Cas13的靶向反式剪接 Download PDF

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CN115023499A
CN115023499A CN202080058066.XA CN202080058066A CN115023499A CN 115023499 A CN115023499 A CN 115023499A CN 202080058066 A CN202080058066 A CN 202080058066A CN 115023499 A CN115023499 A CN 115023499A
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trans
splicing
splicing system
targeted
targeted trans
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Chinese (zh)
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雅各布·博拉霍
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Massachusetts Institute of Technology
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Massachusetts Institute of Technology
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/40—Systems of functionally co-operating vectors

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Medicinal 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)
  • Peptides Or Proteins (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN202080058066.XA 2019-08-16 2020-08-14 使用CRISPR/Cas13的靶向反式剪接 Pending CN115023499A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962888210P 2019-08-16 2019-08-16
US62/888,210 2019-08-16
US202062985633P 2020-03-05 2020-03-05
US62/985,633 2020-03-05
PCT/US2020/046523 WO2021034717A1 (en) 2019-08-16 2020-08-14 Targeted trans-splicing using crispr/cas13

Publications (1)

Publication Number Publication Date
CN115023499A true CN115023499A (zh) 2022-09-06

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ID=72291120

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Application Number Title Priority Date Filing Date
CN202080058066.XA Pending CN115023499A (zh) 2019-08-16 2020-08-14 使用CRISPR/Cas13的靶向反式剪接

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US (3) US11767528B2 (https=)
EP (1) EP4013873A1 (https=)
JP (1) JP2022544688A (https=)
KR (1) KR20220044811A (https=)
CN (1) CN115023499A (https=)
AU (1) AU2020333667A1 (https=)
CA (1) CA3150747A1 (https=)
WO (1) WO2021034717A1 (https=)

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EP4277987A4 (en) * 2021-02-25 2025-07-16 Univ Duke COMPOSITIONS AND METHODS FOR TRANSCRIPTOME ENGINEERING
WO2022187524A1 (en) * 2021-03-05 2022-09-09 The Jackson Laboratory Multiplex rna targeting
WO2022266849A1 (zh) * 2021-06-22 2022-12-29 中国科学院脑科学与智能技术卓越创新中心 新型CRISPR-Cas13蛋白的筛选及其应用
EP4141112B1 (en) * 2021-07-08 2025-06-11 Korea Advanced Institute of Science and Technology Rna expression regulation or editing method through activity control of cas13 protein
AU2022337146A1 (en) 2021-09-03 2024-03-14 Tacit Therapeutics, Inc. Rna editing via recruitment of spliceosome components
AU2022343271A1 (en) * 2021-09-08 2024-03-28 Flagship Pioneering Innovations Vi, Llc Recruitment in trans of gene editing system components
EP4404953A4 (en) * 2021-09-21 2025-09-24 Univ Johns Hopkins MRNA REGULATORY THERAPY FOR THE TREATMENT OF HAPLOINSUFFICIENCY DISORDERS
US20240409920A1 (en) * 2021-10-15 2024-12-12 The Broad Institute, Inc. Rna-guided trans-splicing of rna
EP4522758A4 (en) 2022-05-13 2026-05-06 Ascidian Therapeutics Inc Abca4 trans-splicing molecules
CA3265828A1 (en) * 2022-08-25 2024-02-29 Massachusetts Institute Of Technology PROGRAMMABLE RNA WRITING USING CRISPR EFFECTORS AND TRANS-SPLICING MODELS
WO2024112957A1 (en) 2022-11-23 2024-05-30 Amber Bio Inc. Gene-modifying endonucleases
US20250151704A1 (en) * 2023-11-10 2025-05-15 Joseph Fenton Lawler Trans-splicing methods and compositions for generation of single sex offspring
WO2026068725A1 (en) * 2024-09-26 2026-04-02 Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Synthetic transfer vehicles for delivering rna to cells

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US20140206546A1 (en) * 2013-01-14 2014-07-24 Cellecta, Inc. Methods and compositions for single cell expression profiling
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EP4013873A1 (en) 2022-06-22
US20240035031A1 (en) 2024-02-01
WO2021034717A1 (en) 2021-02-25
US11767528B2 (en) 2023-09-26
AU2020333667A1 (en) 2022-02-24
JP2022544688A (ja) 2022-10-20
US12297433B2 (en) 2025-05-13
US20250236872A1 (en) 2025-07-24
US20210071178A1 (en) 2021-03-11
KR20220044811A (ko) 2022-04-11
CA3150747A1 (en) 2021-02-25

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Application publication date: 20220906