JP7498499B2 - 心臓、骨格筋、及び筋幹細胞におけるインビボ相同組換え修復 - Google Patents

心臓、骨格筋、及び筋幹細胞におけるインビボ相同組換え修復 Download PDF

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JP7498499B2
JP7498499B2 JP2021510297A JP2021510297A JP7498499B2 JP 7498499 B2 JP7498499 B2 JP 7498499B2 JP 2021510297 A JP2021510297 A JP 2021510297A JP 2021510297 A JP2021510297 A JP 2021510297A JP 7498499 B2 JP7498499 B2 JP 7498499B2
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aav
cells
nuclease
composition
muscle
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JP2021522858A (ja
JP2021522858A5 (enExample
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エイミー ジェイ． ウェイガーズ，
ケクシアン ジュ，
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Harvard University
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    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • C12N2510/00—Genetically modified cells
    • 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
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Mycology (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Plant Pathology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2021510297A 2018-05-03 2019-05-03 心臓、骨格筋、及び筋幹細胞におけるインビボ相同組換え修復 Active JP7498499B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024085010A JP2024100980A (ja) 2018-05-03 2024-05-24 心臓、骨格筋、及び筋幹細胞におけるインビボ相同組換え修復

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862666685P 2018-05-03 2018-05-03
US62/666,685 2018-05-03
PCT/US2019/030748 WO2019213626A1 (en) 2018-05-03 2019-05-03 In vivo homology directed repair in heart, skeletal muscle, and muscle stem cells

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JP2024085010A Division JP2024100980A (ja) 2018-05-03 2024-05-24 心臓、骨格筋、及び筋幹細胞におけるインビボ相同組換え修復

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JP2021522858A JP2021522858A (ja) 2021-09-02
JP2021522858A5 JP2021522858A5 (enExample) 2022-05-10
JP7498499B2 true JP7498499B2 (ja) 2024-06-12

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JP2021510297A Active JP7498499B2 (ja) 2018-05-03 2019-05-03 心臓、骨格筋、及び筋幹細胞におけるインビボ相同組換え修復
JP2024085010A Pending JP2024100980A (ja) 2018-05-03 2024-05-24 心臓、骨格筋、及び筋幹細胞におけるインビボ相同組換え修復

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US (1) US20210363546A1 (enExample)
EP (1) EP3787692A4 (enExample)
JP (2) JP7498499B2 (enExample)
CN (1) CN112512595A (enExample)
AU (1) AU2019262225B2 (enExample)
CA (1) CA3099332A1 (enExample)
WO (1) WO2019213626A1 (enExample)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
US12214054B2 (en) 2015-11-30 2025-02-04 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
EP3443081A4 (en) 2016-04-13 2019-10-30 Duke University CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE
JP7490211B2 (ja) 2016-07-19 2024-05-27 デューク ユニバーシティ Ｃｐｆ１に基づくゲノム編集の治療適用
US12509492B2 (en) 2018-01-19 2025-12-30 Duke University Genome engineering with CRISPR-Cas systems in eukaryotes
MX2021001850A (es) 2018-08-18 2021-07-15 Harvard College Edicion genica in situ.
TWI887234B (zh) 2019-04-14 2025-06-21 美國公爵大學 治療杜興氏（ｄｕｃｈｅｎｎｅ）肌肉失養症之大型突變熱點之ａａｖ載體介導之刪除
EP4125349A4 (en) * 2020-04-27 2024-07-10 Duke University GENE EDITING OF SATELLITE CELLS IN VIVO USING AAV VECTORS ENCODING MUSCLE-SPECIFIC PROMOTERS
CN115181722B (zh) * 2022-08-30 2024-06-11 江苏农牧科技职业学院 一种鹅骨骼肌卫星细胞的体外分离和培养方法
WO2024229028A2 (en) * 2023-05-01 2024-11-07 University Of Washington Novel regulatory cassettes for specific expression of genes in muscle stem cells
WO2025260086A1 (en) * 2024-06-14 2025-12-18 President And Fellows Of Harvard College In vivo site-specific base editing

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017525696A (ja) 2014-08-11 2017-09-07 ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム Ｃｒｉｓｐｒ／ｃａｓ９媒介遺伝子編集による筋ジストロフィーの予防
WO2018013932A1 (en) 2016-07-15 2018-01-18 Salk Institute For Biological Studies Methods and compositions for genome editing in non-dividing cells

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG10201707569YA (en) * 2012-12-12 2017-10-30 Broad Inst Inc Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications
US20170362635A1 (en) * 2016-06-20 2017-12-21 University Of Washington Muscle-specific crispr/cas9 editing of genes
WO2018058064A1 (en) * 2016-09-23 2018-03-29 Casebia Therapeutics Limited Liability Partnership Compositions and methods for gene editing

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017525696A (ja) 2014-08-11 2017-09-07 ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム Ｃｒｉｓｐｒ／ｃａｓ９媒介遺伝子編集による筋ジストロフィーの予防
WO2018013932A1 (en) 2016-07-15 2018-01-18 Salk Institute For Biological Studies Methods and compositions for genome editing in non-dividing cells

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Scientific Report，2017年，7:9363

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Publication number Publication date
EP3787692A4 (en) 2022-03-23
CN112512595A (zh) 2021-03-16
JP2024100980A (ja) 2024-07-26
JP2021522858A (ja) 2021-09-02
CA3099332A1 (en) 2019-11-07
AU2019262225B2 (en) 2025-08-21
US20210363546A1 (en) 2021-11-25
EP3787692A1 (en) 2021-03-10
AU2019262225A1 (en) 2021-01-07
WO2019213626A1 (en) 2019-11-07

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