CN112512595A - 心脏、骨骼肌和肌肉干细胞中的体内同源性定向修复 - Google Patents

心脏、骨骼肌和肌肉干细胞中的体内同源性定向修复 Download PDF

Info

Publication number
CN112512595A
CN112512595A CN201980042149.7A CN201980042149A CN112512595A CN 112512595 A CN112512595 A CN 112512595A CN 201980042149 A CN201980042149 A CN 201980042149A CN 112512595 A CN112512595 A CN 112512595A
Authority
CN
China
Prior art keywords
nuclease
muscle
virus
cell
cells
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
CN201980042149.7A
Other languages
English (en)
Chinese (zh)
Inventor
艾米·J·韦格斯
朱克先
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.)
Harvard University
Original Assignee
Harvard University
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 Harvard University filed Critical Harvard University
Publication of CN112512595A publication Critical patent/CN112512595A/zh
Pending legal-status Critical Current

Links

Images

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
    • C12N15/87—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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Mycology (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)
CN201980042149.7A 2018-05-03 2019-05-03 心脏、骨骼肌和肌肉干细胞中的体内同源性定向修复 Pending CN112512595A (zh)

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

Publications (1)

Publication Number Publication Date
CN112512595A true CN112512595A (zh) 2021-03-16

Family

ID=68386147

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201980042149.7A Pending CN112512595A (zh) 2018-05-03 2019-05-03 心脏、骨骼肌和肌肉干细胞中的体内同源性定向修复

Country Status (7)

Country Link
US (1) US20210363546A1 (https=)
EP (1) EP3787692A4 (https=)
JP (2) JP7498499B2 (https=)
CN (1) CN112512595A (https=)
AU (1) AU2019262225B2 (https=)
CA (1) CA3099332A1 (https=)
WO (1) WO2019213626A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115181722A (zh) * 2022-08-30 2022-10-14 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

Families Citing this family (10)

* 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
EA201891317A3 (ru) 2015-11-30 2019-04-30 Дьюк Юниверсити Терапевтические мишени для коррекции гена дистрофина человека с помощью редактирования генов и способы их применения
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
WO2018017754A1 (en) 2016-07-19 2018-01-25 Duke University Therapeutic applications of cpf1-based genome editing
WO2019144061A1 (en) 2018-01-19 2019-07-25 Duke University Genome engineering with crispr-cas systems in eukaryotes
US12486519B2 (en) 2018-08-18 2025-12-02 President And Fellows Of Harvard College In situ gene editing
EP4125349A4 (en) * 2020-04-27 2024-07-10 Duke University GENE EDITING OF SATELLITE CELLS IN VIVO USING AAV VECTORS ENCODING MUSCLE-SPECIFIC PROMOTERS
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 (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN106714845A (zh) * 2014-08-11 2017-05-24 得克萨斯州大学系统董事会 通过crispr/cas9介导的基因编辑预防肌营养不良
US20170362635A1 (en) * 2016-06-20 2017-12-21 University Of Washington Muscle-specific crispr/cas9 editing of genes
WO2018013932A1 (en) * 2016-07-15 2018-01-18 Salk Institute For Biological Studies Methods and compositions for genome editing in non-dividing cells
WO2018058064A1 (en) * 2016-09-23 2018-03-29 Casebia Therapeutics Limited Liability Partnership Compositions and methods for gene editing

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2931897T3 (en) * 2012-12-12 2018-02-05 Broad Inst Inc CONSTRUCTION, MODIFICATION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION AND THERAPEUTICAL APPLICATIONS

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN106714845A (zh) * 2014-08-11 2017-05-24 得克萨斯州大学系统董事会 通过crispr/cas9介导的基因编辑预防肌营养不良
US20170362635A1 (en) * 2016-06-20 2017-12-21 University Of Washington Muscle-specific crispr/cas9 editing of genes
WO2018013932A1 (en) * 2016-07-15 2018-01-18 Salk Institute For Biological Studies Methods and compositions for genome editing in non-dividing cells
WO2018058064A1 (en) * 2016-09-23 2018-03-29 Casebia Therapeutics Limited Liability Partnership Compositions and methods for gene editing

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
KATSUYA INAGAKI等: "Robust Systemic Transduction with AAV9 Vectors in Mice: Efficient Global Cardiac Gene Transfer Superior to That of AAV8", MOL THER., vol. 14, no. 1, pages 45 *
TAKAMARU ISHIZU等: "Targeted Genome Replacement via Homology-directed Repair in Non-dividing Cardiomyocytes", SCIENTIFIC REPORTS, vol. 7, pages 9363 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115181722A (zh) * 2022-08-30 2022-10-14 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
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

Also Published As

Publication number Publication date
JP7498499B2 (ja) 2024-06-12
EP3787692A1 (en) 2021-03-10
US20210363546A1 (en) 2021-11-25
CA3099332A1 (en) 2019-11-07
JP2021522858A (ja) 2021-09-02
WO2019213626A1 (en) 2019-11-07
JP2024100980A (ja) 2024-07-26
AU2019262225A1 (en) 2021-01-07
AU2019262225B2 (en) 2025-08-21
EP3787692A4 (en) 2022-03-23

Similar Documents

Publication Publication Date Title
JP7498499B2 (ja) 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
CN114174520B (zh) 用于选择性基因调节的组合物和方法
JP6683691B2 (ja) 高効率ゲノム編集のためのアデノ随伴ウイルスベクターバリアント及びその方法
KR20250096876A (ko) 안과 질환을 위한 세포 모델 및 치료요법
JP2021511803A (ja) ヒト心筋細胞におけるジストロフィン変異を修正するための組成物および方法
JP2023515710A (ja) ＣＲＩＳＰＲ媒介式エクソン欠失用の最適なｇＲＮＡ対を発見するためのハイスループットスクリーニング法
EP3262065B1 (en) Methods and compositions for treating dystroglycanopathy disorders
CN111718420A (zh) 一种用于基因治疗的融合蛋白及其应用
US20230272428A1 (en) Methods and compositions for correction of dmd mutations
US20250304955A1 (en) Compositions and methods for myosin heavy chain base editing
CN117980482A (zh) Rbm20突变的基因组编辑
CN117561331A (zh) 用于产生经基因修饰的原代细胞的方法和组合物
Wong Utilization of CRISPR/Cas9-mediated gene editing for correction of deletion mutations in DMD
TWI838812B (zh) 用於治療法布瑞氏症之組合物及方法
CN105358690B (zh) 启动子组合物
US12486519B2 (en) In situ gene editing
KR20240027748A (ko) Rbm20 돌연변이의 게놈 편집
WO2026005012A1 (ja) インビトロ及びインビボのゲノム編集における相同組換えの効率を高める方法
KR20250141243A (ko) 데그론 융합 단백질 및 그의 생산 및 사용 방법
CN119013408A (zh) 用于致心律失常性心肌病的基因疗法
WO2020187272A1 (zh) 一种用于基因治疗的融合蛋白及其应用
BR122024014087A2 (pt) Vetores e células

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination