CA3099332A1 - In vivo homology directed repair in heart, skeletal muscle, and muscle stem cells - Google Patents
In vivo homology directed repair in heart, skeletal muscle, and muscle stem cells Download PDFInfo
- Publication number
- CA3099332A1 CA3099332A1 CA3099332A CA3099332A CA3099332A1 CA 3099332 A1 CA3099332 A1 CA 3099332A1 CA 3099332 A CA3099332 A CA 3099332A CA 3099332 A CA3099332 A CA 3099332A CA 3099332 A1 CA3099332 A1 CA 3099332A1
- Authority
- CA
- Canada
- Prior art keywords
- virus
- muscle
- nuclease
- cells
- cell
- 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
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Classifications
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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
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- 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
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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
- 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)
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 |
|---|---|
| CA3099332A1 true CA3099332A1 (en) | 2019-11-07 |
Family
ID=68386147
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3099332A Pending CA3099332A1 (en) | 2018-05-03 | 2019-05-03 | In vivo homology directed repair in heart, skeletal muscle, and muscle stem cells |
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=) |
Families Citing this family (11)
| 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 |
| 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 |
Family Cites Families (5)
| 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 |
| WO2016025469A1 (en) * | 2014-08-11 | 2016-02-18 | The Board Of Regents Of The University Of Texas System | Prevention of muscular dystrophy by crispr/cas9-mediated gene editing |
| 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 |
| EP3516058A1 (en) * | 2016-09-23 | 2019-07-31 | Casebia Therapeutics Limited Liability Partnership | Compositions and methods for gene editing |
-
2019
- 2019-05-03 WO PCT/US2019/030748 patent/WO2019213626A1/en not_active Ceased
- 2019-05-03 CN CN201980042149.7A patent/CN112512595A/zh active Pending
- 2019-05-03 AU AU2019262225A patent/AU2019262225B2/en active Active
- 2019-05-03 EP EP19796061.0A patent/EP3787692A4/en active Pending
- 2019-05-03 JP JP2021510297A patent/JP7498499B2/ja active Active
- 2019-05-03 CA CA3099332A patent/CA3099332A1/en active Pending
- 2019-05-03 US US17/052,798 patent/US20210363546A1/en active Pending
-
2024
- 2024-05-24 JP JP2024085010A patent/JP2024100980A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP7498499B2 (ja) | 2024-06-12 |
| EP3787692A1 (en) | 2021-03-10 |
| US20210363546A1 (en) | 2021-11-25 |
| 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 |
| CN112512595A (zh) | 2021-03-16 |
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