CA3218209A1 - Multiplex crispr/cas9-mediated target gene activation system - Google Patents

Multiplex crispr/cas9-mediated target gene activation system Download PDF

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Publication number
CA3218209A1
CA3218209A1 CA3218209A CA3218209A CA3218209A1 CA 3218209 A1 CA3218209 A1 CA 3218209A1 CA 3218209 A CA3218209 A CA 3218209A CA 3218209 A CA3218209 A CA 3218209A CA 3218209 A1 CA3218209 A1 CA 3218209A1
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Prior art keywords
nucleic acid
seq
sequence
modified
sgrna
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CA3218209A
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English (en)
French (fr)
Inventor
Juan Carlos Izpisua Belmonte
Chao Wang
Hsin-Kai Liao
Pradeep REDDY
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Salk Institute for Biological Studies
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Salk Institute for Biological Studies
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    • 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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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6806—Preparing nucleic acids for analysis, e.g. for polymerase chain reaction [PCR] assay
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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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
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    • C12N2320/00—Applications; Uses
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    • C12N2330/00—Production
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    • 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

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  • Health & Medical Sciences (AREA)
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  • Wood Science & Technology (AREA)
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  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
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  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Analytical Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Breeding Of Plants And Reproduction By Means Of Culturing (AREA)
  • Peptides Or Proteins (AREA)
CA3218209A 2021-04-28 2022-04-28 Multiplex crispr/cas9-mediated target gene activation system Pending CA3218209A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163181059P 2021-04-28 2021-04-28
US63/181,059 2021-04-28
PCT/US2022/026805 WO2022232442A2 (en) 2021-04-28 2022-04-28 Multiplex crispr/cas9-mediated target gene activation system

Publications (1)

Publication Number Publication Date
CA3218209A1 true CA3218209A1 (en) 2022-11-03

Family

ID=83848892

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3218209A Pending CA3218209A1 (en) 2021-04-28 2022-04-28 Multiplex crispr/cas9-mediated target gene activation system

Country Status (7)

Country Link
US (1) US20240209354A1 (https=)
EP (1) EP4330375A4 (https=)
JP (1) JP2024515827A (https=)
CN (1) CN117580941A (https=)
AU (1) AU2022267320A1 (https=)
CA (1) CA3218209A1 (https=)
WO (1) WO2022232442A2 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025111340A1 (en) * 2023-11-20 2025-05-30 William Marsh Rice University Engineered extrachromosomal nucleic acids and methods of use thereof
WO2025227064A1 (en) * 2024-04-26 2025-10-30 Renasci Biotechnologies, Inc. Genomic editing methods to treat cardiovascular disease, and compositions for use in practicing the same
CN119020323A (zh) * 2024-09-29 2024-11-26 中国农业科学院作物科学研究所 一种融合外切酶的CRIPSR/Cas12i3小麦基因编辑体系

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6793547B2 (ja) * 2013-12-12 2020-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能ＣＲＩＳＰＲ−Ｃａｓ系による配列操作のための系、方法および組成物
AU2017219605B2 (en) * 2016-02-15 2023-04-13 Temple University - Of The Commonwealth System Of Higher Education Excision of retroviral nucleic acid sequences
AU2018393050A1 (en) * 2017-12-21 2020-06-18 Bayer Healthcare Llc Materials and methods for treatment of Usher Syndrome Type 2A
WO2019236081A1 (en) * 2018-06-06 2019-12-12 Salk Institute For Biological Studies Targeted gene activation using modified guide rna
EP4028063A1 (en) * 2019-09-13 2022-07-20 Regeneron Pharmaceuticals, Inc. Transcription modulation in animals using crispr/cas systems delivered by lipid nanoparticles

Also Published As

Publication number Publication date
WO2022232442A3 (en) 2022-12-15
AU2022267320A9 (en) 2023-11-30
EP4330375A2 (en) 2024-03-06
WO2022232442A2 (en) 2022-11-03
US20240209354A1 (en) 2024-06-27
AU2022267320A1 (en) 2023-11-23
JP2024515827A (ja) 2024-04-10
EP4330375A4 (en) 2025-08-13
CN117580941A (zh) 2024-02-20

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