AU2022267320A1 - 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
AU2022267320A1
AU2022267320A1 AU2022267320A AU2022267320A AU2022267320A1 AU 2022267320 A1 AU2022267320 A1 AU 2022267320A1 AU 2022267320 A AU2022267320 A AU 2022267320A AU 2022267320 A AU2022267320 A AU 2022267320A AU 2022267320 A1 AU2022267320 A1 AU 2022267320A1
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nucleic acid
seq
sequence
modified
sgrna
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AU2022267320A
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AU2022267320A9 (en
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Juan Carlos Izpisua Belmonte
Hsin-Kai Liao
Pradeep REDDY
Chao Wang
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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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    • 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
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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
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    • C12N2320/00—Applications; Uses
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    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
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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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  • 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)
AU2022267320A 2021-04-28 2022-04-28 Multiplex crispr/cas9-mediated target gene activation system Pending AU2022267320A1 (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 (2)

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AU2022267320A1 true AU2022267320A1 (en) 2023-11-23
AU2022267320A9 AU2022267320A9 (en) 2023-11-30

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AU2022267320A Pending AU2022267320A1 (en) 2021-04-28 2022-04-28 Multiplex crispr/cas9-mediated target gene activation system

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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

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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
JP2024515827A (ja) 2024-04-10
CA3218209A1 (en) 2022-11-03
EP4330375A4 (en) 2025-08-13
CN117580941A (zh) 2024-02-20

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