JP2025016714A5 - - Google Patents
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- JP2025016714A5 JP2025016714A5 JP2024193935A JP2024193935A JP2025016714A5 JP 2025016714 A5 JP2025016714 A5 JP 2025016714A5 JP 2024193935 A JP2024193935 A JP 2024193935A JP 2024193935 A JP2024193935 A JP 2024193935A JP 2025016714 A5 JP2025016714 A5 JP 2025016714A5
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- 230000008685 targeting Effects 0.000 claims 18
- 108010042407 Endonucleases Proteins 0.000 claims 16
- 102000004533 Endonucleases Human genes 0.000 claims 16
- 230000003612 virological effect Effects 0.000 claims 16
- 238000000034 method Methods 0.000 claims 14
- 238000003776 cleavage reaction Methods 0.000 claims 8
- 230000007017 scission Effects 0.000 claims 8
- 101710163270 Nuclease Proteins 0.000 claims 7
- 108090000623 proteins and genes Proteins 0.000 claims 6
- 108091033409 CRISPR Proteins 0.000 claims 5
- 108091034117 Oligonucleotide Proteins 0.000 claims 4
- 230000035772 mutation Effects 0.000 claims 4
- 238000010354 CRISPR gene editing Methods 0.000 claims 3
- 108020004414 DNA Proteins 0.000 claims 3
- 208000026350 Inborn Genetic disease Diseases 0.000 claims 3
- 208000016361 genetic disease Diseases 0.000 claims 3
- 108020004707 nucleic acids Proteins 0.000 claims 3
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- 150000007523 nucleic acids Chemical class 0.000 claims 3
- 239000013612 plasmid Substances 0.000 claims 3
- 230000001177 retroviral effect Effects 0.000 claims 3
- 208000011580 syndromic disease Diseases 0.000 claims 3
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- 201000003533 Leber congenital amaurosis Diseases 0.000 claims 2
- 208000007014 Retinitis pigmentosa Diseases 0.000 claims 2
- 208000027073 Stargardt disease Diseases 0.000 claims 2
- 201000007960 WAGR syndrome Diseases 0.000 claims 2
- 210000001789 adipocyte Anatomy 0.000 claims 2
- 210000002457 barrier cell Anatomy 0.000 claims 2
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- 238000001727 in vivo Methods 0.000 claims 2
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- 102100028187 ATP-binding cassette sub-family C member 6 Human genes 0.000 claims 1
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- 206010001557 Albinism Diseases 0.000 claims 1
- 101100385358 Alicyclobacillus acidoterrestris (strain ATCC 49025 / DSM 3922 / CIP 106132 / NCIMB 13137 / GD3B) cas12b gene Proteins 0.000 claims 1
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- 208000010061 Autosomal Dominant Polycystic Kidney Diseases 0.000 claims 1
- 210000002237 B-cell of pancreatic islet Anatomy 0.000 claims 1
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- 101150074775 Csf1 gene Proteins 0.000 claims 1
- 201000003883 Cystic fibrosis Diseases 0.000 claims 1
- 102000053602 DNA Human genes 0.000 claims 1
- 201000010374 Down Syndrome Diseases 0.000 claims 1
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- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims 1
- 108700024394 Exon Proteins 0.000 claims 1
- 206010016207 Familial Mediterranean fever Diseases 0.000 claims 1
- 241000282326 Felis catus Species 0.000 claims 1
- 208000001914 Fragile X syndrome Diseases 0.000 claims 1
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- 206010020365 Homocystinuria Diseases 0.000 claims 1
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- 102100024640 Low-density lipoprotein receptor Human genes 0.000 claims 1
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- 241000283973 Oryctolagus cuniculus Species 0.000 claims 1
- 206010031243 Osteogenesis imperfecta Diseases 0.000 claims 1
- 208000018737 Parkinson disease Diseases 0.000 claims 1
- 241001494479 Pecora Species 0.000 claims 1
- 201000011252 Phenylketonuria Diseases 0.000 claims 1
- 208000019222 Poland syndrome Diseases 0.000 claims 1
- 241000097929 Porphyria Species 0.000 claims 1
- 208000010642 Porphyrias Diseases 0.000 claims 1
- 208000007932 Progeria Diseases 0.000 claims 1
- 206010060862 Prostate cancer Diseases 0.000 claims 1
- 208000000236 Prostatic Neoplasms Diseases 0.000 claims 1
- 201000004613 Pseudoxanthoma elasticum Diseases 0.000 claims 1
- 208000017442 Retinal disease Diseases 0.000 claims 1
- 201000000582 Retinoblastoma Diseases 0.000 claims 1
- 208000000453 Skin Neoplasms Diseases 0.000 claims 1
- 208000027077 Stickler syndrome Diseases 0.000 claims 1
- 210000001744 T-lymphocyte Anatomy 0.000 claims 1
- 238000010459 TALEN Methods 0.000 claims 1
- 208000002903 Thalassemia Diseases 0.000 claims 1
- 108010043645 Transcription Activator-Like Effector Nucleases Proteins 0.000 claims 1
- 206010068233 Trimethylaminuria Diseases 0.000 claims 1
- 206010044688 Trisomy 21 Diseases 0.000 claims 1
- 208000026928 Turner syndrome Diseases 0.000 claims 1
- 206010045261 Type IIa hyperlipidaemia Diseases 0.000 claims 1
- 208000014769 Usher Syndromes Diseases 0.000 claims 1
- 201000002916 Warburg micro syndrome Diseases 0.000 claims 1
- 208000018839 Wilson disease Diseases 0.000 claims 1
- 201000006083 Xeroderma Pigmentosum Diseases 0.000 claims 1
- 108010017070 Zinc Finger Nucleases Proteins 0.000 claims 1
- 208000008919 achondroplasia Diseases 0.000 claims 1
- 208000006682 alpha 1-Antitrypsin Deficiency Diseases 0.000 claims 1
- 208000022185 autosomal dominant polycystic kidney disease Diseases 0.000 claims 1
- 210000003719 b-lymphocyte Anatomy 0.000 claims 1
- 210000003651 basophil Anatomy 0.000 claims 1
- 210000002449 bone cell Anatomy 0.000 claims 1
- 201000011510 cancer Diseases 0.000 claims 1
- 210000004413 cardiac myocyte Anatomy 0.000 claims 1
- 208000029742 colonic neoplasm Diseases 0.000 claims 1
- 230000037430 deletion Effects 0.000 claims 1
- 238000012217 deletion Methods 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 230000034431 double-strand break repair via homologous recombination Effects 0.000 claims 1
- 210000003979 eosinophil Anatomy 0.000 claims 1
- 108010091897 factor V Leiden Proteins 0.000 claims 1
- 201000001386 familial hypercholesterolemia Diseases 0.000 claims 1
- 230000002440 hepatic effect Effects 0.000 claims 1
- 210000003494 hepatocyte Anatomy 0.000 claims 1
- 210000003630 histaminocyte Anatomy 0.000 claims 1
- 208000009624 holoprosencephaly Diseases 0.000 claims 1
- 239000005556 hormone Substances 0.000 claims 1
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- 230000037431 insertion Effects 0.000 claims 1
- 238000003780 insertion Methods 0.000 claims 1
- 210000004698 lymphocyte Anatomy 0.000 claims 1
- 210000001616 monocyte Anatomy 0.000 claims 1
- 210000000822 natural killer cell Anatomy 0.000 claims 1
- 201000004931 neurofibromatosis Diseases 0.000 claims 1
- 210000004498 neuroglial cell Anatomy 0.000 claims 1
- 210000002569 neuron Anatomy 0.000 claims 1
- 210000000440 neutrophil Anatomy 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
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- 210000001915 nurse cell Anatomy 0.000 claims 1
- 210000000287 oocyte Anatomy 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 210000004180 plasmocyte Anatomy 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 208000023558 pseudoxanthoma elasticum (inherited or acquired) Diseases 0.000 claims 1
- 230000003248 secreting effect Effects 0.000 claims 1
- 210000002955 secretory cell Anatomy 0.000 claims 1
- 208000007056 sickle cell anemia Diseases 0.000 claims 1
- 210000002363 skeletal muscle cell Anatomy 0.000 claims 1
- 201000000849 skin cancer Diseases 0.000 claims 1
- 210000000329 smooth muscle myocyte Anatomy 0.000 claims 1
- 208000002320 spinal muscular atrophy Diseases 0.000 claims 1
- 210000000130 stem cell Anatomy 0.000 claims 1
- 238000006467 substitution reaction Methods 0.000 claims 1
- 201000005665 thrombophilia Diseases 0.000 claims 1
- 230000005945 translocation Effects 0.000 claims 1
- 230000002485 urinary effect Effects 0.000 claims 1
- 208000006542 von Hippel-Lindau disease Diseases 0.000 claims 1
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962890542P | 2019-08-22 | 2019-08-22 | |
| US62/890,542 | 2019-08-22 | ||
| US201962891210P | 2019-08-23 | 2019-08-23 | |
| US62/891,210 | 2019-08-23 | ||
| JP2022511242A JP7628319B2 (ja) | 2019-08-22 | 2019-09-20 | in vivo遺伝子編集のための組成物および方法 |
| PCT/US2019/052266 WO2021034336A1 (en) | 2019-08-22 | 2019-09-20 | Compositions and methods for in vivo gene editing |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022511242A Division JP7628319B2 (ja) | 2019-08-22 | 2019-09-20 | in vivo遺伝子編集のための組成物および方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2025016714A JP2025016714A (ja) | 2025-02-04 |
| JP2025016714A5 true JP2025016714A5 (enExample) | 2025-03-17 |
Family
ID=74660737
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022511242A Active JP7628319B2 (ja) | 2019-08-22 | 2019-09-20 | in vivo遺伝子編集のための組成物および方法 |
| JP2024193935A Pending JP2025016714A (ja) | 2019-08-22 | 2024-11-05 | in vivo遺伝子編集のための組成物および方法 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022511242A Active JP7628319B2 (ja) | 2019-08-22 | 2019-09-20 | in vivo遺伝子編集のための組成物および方法 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20220333106A1 (enExample) |
| EP (1) | EP4017543A4 (enExample) |
| JP (2) | JP7628319B2 (enExample) |
| AU (1) | AU2019462506A1 (enExample) |
| CA (1) | CA3152056A1 (enExample) |
| WO (1) | WO2021034336A1 (enExample) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20220333106A1 (en) * | 2019-08-22 | 2022-10-20 | Salk Institute For Biological Studies | Compositions and methods for in vivo gene editing |
| CN116200368A (zh) * | 2021-11-30 | 2023-06-02 | 上海科技大学 | 一种基于c2c9核酸酶的新型基因组编辑系统及其应用 |
| WO2023187779A1 (en) * | 2022-03-28 | 2023-10-05 | Ramot At Tel-Aviv University Ltd. | Site-specific in vivo t cell engineering, systems, compositions and methods thereof |
| CN118006739B (zh) * | 2024-02-05 | 2024-10-29 | 北京亿森宝生物科技有限公司 | 一种同时检测禽滑液囊支原体野毒和疫苗的方法、产品及应用 |
| CN119685398B (zh) * | 2025-02-26 | 2025-05-23 | 北京林业大学 | 一种敲除牛fbxo40基因的方法及其应用 |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20120196370A1 (en) * | 2010-12-03 | 2012-08-02 | Fyodor Urnov | Methods and compositions for targeted genomic deletion |
| US11939571B2 (en) * | 2016-06-10 | 2024-03-26 | President And Fellows Of Harvard College | Library-scale engineering of metabolic pathways |
| US11674158B2 (en) * | 2016-07-15 | 2023-06-13 | Salk Institute For Biological Studies | Methods and compositions for genome editing in non-dividing cells |
| US20220333106A1 (en) * | 2019-08-22 | 2022-10-20 | Salk Institute For Biological Studies | Compositions and methods for in vivo gene editing |
-
2019
- 2019-09-20 US US17/637,058 patent/US20220333106A1/en active Pending
- 2019-09-20 AU AU2019462506A patent/AU2019462506A1/en active Pending
- 2019-09-20 WO PCT/US2019/052266 patent/WO2021034336A1/en not_active Ceased
- 2019-09-20 EP EP19942111.6A patent/EP4017543A4/en active Pending
- 2019-09-20 JP JP2022511242A patent/JP7628319B2/ja active Active
- 2019-09-20 CA CA3152056A patent/CA3152056A1/en active Pending
-
2024
- 2024-11-05 JP JP2024193935A patent/JP2025016714A/ja active Pending
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