CA3150747A1 - Targeted trans-splicing using crispr/cas13 - Google Patents

Targeted trans-splicing using crispr/cas13

Info

Publication number
CA3150747A1
CA3150747A1 CA3150747A CA3150747A CA3150747A1 CA 3150747 A1 CA3150747 A1 CA 3150747A1 CA 3150747 A CA3150747 A CA 3150747A CA 3150747 A CA3150747 A CA 3150747A CA 3150747 A1 CA3150747 A1 CA 3150747A1
Authority
CA
Canada
Prior art keywords
trans
splicing
splicing system
targeted
targeted trans
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
CA3150747A
Other languages
English (en)
French (fr)
Inventor
Jacob BORRAJO
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Massachusetts Institute of Technology
Original Assignee
Massachusetts Institute of Technology
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Massachusetts Institute of Technology filed Critical Massachusetts Institute of Technology
Publication of CA3150747A1 publication Critical patent/CA3150747A1/en
Pending legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • 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
    • C12N15/63—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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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

Landscapes

  • 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)
CA3150747A 2019-08-16 2020-08-14 Targeted trans-splicing using crispr/cas13 Pending CA3150747A1 (en)

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
CA3150747A1 true CA3150747A1 (en) 2021-02-25

Family

ID=72291120

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3150747A Pending CA3150747A1 (en) 2019-08-16 2020-08-14 Targeted trans-splicing using crispr/cas13

Country Status (8)

Country Link
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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US4186183A (en) 1978-03-29 1980-01-29 The United States Of America As Represented By The Secretary Of The Army Liposome carriers in chemotherapy of leishmaniasis
US4261975A (en) 1979-09-19 1981-04-14 Merck & Co., Inc. Viral liposome particle
US4554101A (en) 1981-01-09 1985-11-19 New York Blood Center, Inc. Identification and preparation of epitopes on antigens and allergens on the basis of hydrophilicity
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Also Published As

Publication number Publication date
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
CN115023499A (zh) 2022-09-06
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

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