JPWO2019213626A5 - - Google Patents
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- Publication number
- JPWO2019213626A5 JPWO2019213626A5 JP2021510297A JP2021510297A JPWO2019213626A5 JP WO2019213626 A5 JPWO2019213626 A5 JP WO2019213626A5 JP 2021510297 A JP2021510297 A JP 2021510297A JP 2021510297 A JP2021510297 A JP 2021510297A JP WO2019213626 A5 JPWO2019213626 A5 JP WO2019213626A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- nuclease
- donor template
- cells
- muscle
- 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.)
- Granted
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- 241000700605 Viruses Species 0.000 claims description 48
- 238000000034 method Methods 0.000 claims description 40
- 101710163270 Nuclease Proteins 0.000 claims description 38
- 230000008685 targeting Effects 0.000 claims description 26
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 24
- 150000007523 nucleic acids Chemical group 0.000 claims description 24
- 210000002064 heart cell Anatomy 0.000 claims description 20
- 239000002773 nucleotide Substances 0.000 claims description 20
- 125000003729 nucleotide group Chemical group 0.000 claims description 20
- 210000000130 stem cell Anatomy 0.000 claims description 20
- 210000003205 muscle Anatomy 0.000 claims description 18
- 230000002463 transducing effect Effects 0.000 claims description 16
- 108010017070 Zinc Finger Nucleases Proteins 0.000 claims description 8
- 210000004027 cell Anatomy 0.000 claims description 8
- 230000002107 myocardial effect Effects 0.000 claims description 8
- 108020005004 Guide RNA Proteins 0.000 claims description 6
- 238000001727 in vivo Methods 0.000 claims description 6
- 230000004048 modification Effects 0.000 claims description 6
- 238000012986 modification Methods 0.000 claims description 6
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims description 4
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 4
- 241000649045 Adeno-associated virus 10 Species 0.000 claims description 4
- 241000649044 Adeno-associated virus 9 Species 0.000 claims description 4
- 108091033409 CRISPR Proteins 0.000 claims description 4
- 230000006820 DNA synthesis Effects 0.000 claims description 4
- 210000004413 cardiac myocyte Anatomy 0.000 claims description 4
- 239000012636 effector Substances 0.000 claims description 4
- 239000012634 fragment Substances 0.000 claims description 4
- 238000010362 genome editing Methods 0.000 claims description 4
- 238000003780 insertion Methods 0.000 claims description 4
- 230000037431 insertion Effects 0.000 claims description 4
- 210000000663 muscle cell Anatomy 0.000 claims description 4
- 230000002062 proliferating effect Effects 0.000 claims description 4
- 230000035755 proliferation Effects 0.000 claims description 4
- 210000003699 striated muscle Anatomy 0.000 claims description 4
- 210000002948 striated muscle cell Anatomy 0.000 claims description 4
- 230000002103 transcriptional effect Effects 0.000 claims description 4
- 108020004414 DNA Proteins 0.000 claims description 2
- 230000000747 cardiac effect Effects 0.000 claims description 2
- 230000034431 double-strand break repair via homologous recombination Effects 0.000 claims description 2
- 230000003511 endothelial effect Effects 0.000 claims description 2
- 210000005003 heart tissue Anatomy 0.000 claims description 2
- 210000001665 muscle stem cell Anatomy 0.000 claims description 2
- 210000001087 myotubule Anatomy 0.000 claims description 2
- 239000002243 precursor Substances 0.000 claims description 2
- 230000003362 replicative effect Effects 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 37
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 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2021522858A JP2021522858A (ja) | 2021-09-02 |
| JPWO2019213626A5 true JPWO2019213626A5 (https=) | 2022-05-10 |
| JP2021522858A5 JP2021522858A5 (https=) | 2022-05-10 |
| JP7498499B2 JP7498499B2 (ja) | 2024-06-12 |
Family
ID=68386147
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
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
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