JP2018513682A5 - - Google Patents
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- JP2018513682A5 JP2018513682A5 JP2017552447A JP2017552447A JP2018513682A5 JP 2018513682 A5 JP2018513682 A5 JP 2018513682A5 JP 2017552447 A JP2017552447 A JP 2017552447A JP 2017552447 A JP2017552447 A JP 2017552447A JP 2018513682 A5 JP2018513682 A5 JP 2018513682A5
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- 125000003729 nucleotide group Chemical group 0.000 claims description 105
- 238000000034 method Methods 0.000 claims description 101
- 239000002773 nucleotide Substances 0.000 claims description 76
- 210000004027 cell Anatomy 0.000 claims description 35
- 229920001184 polypeptide Polymers 0.000 claims description 35
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 35
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 35
- 108020004707 nucleic acids Proteins 0.000 claims description 24
- 102000039446 nucleic acids Human genes 0.000 claims description 24
- 150000007523 nucleic acids Chemical class 0.000 claims description 24
- 108090000623 proteins and genes Proteins 0.000 claims description 22
- 108020004414 DNA Proteins 0.000 claims description 14
- 230000004048 modification Effects 0.000 claims description 13
- 238000012986 modification Methods 0.000 claims description 13
- 230000033228 biological regulation Effects 0.000 claims description 8
- 125000000548 ribosyl group Chemical group C1([C@H](O)[C@H](O)[C@H](O1)CO)* 0.000 claims description 8
- 108091033409 CRISPR Proteins 0.000 claims description 6
- 230000035772 mutation Effects 0.000 claims description 6
- 210000000130 stem cell Anatomy 0.000 claims description 6
- 108020005004 Guide RNA Proteins 0.000 claims description 5
- 230000000694 effects Effects 0.000 claims description 5
- 108020004999 messenger RNA Proteins 0.000 claims description 5
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 4
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 4
- 210000002865 immune cell Anatomy 0.000 claims description 4
- 230000006780 non-homologous end joining Effects 0.000 claims description 4
- 210000005105 peripheral blood lymphocyte Anatomy 0.000 claims description 4
- 210000003819 peripheral blood mononuclear cell Anatomy 0.000 claims description 4
- 125000002467 phosphate group Chemical group [H]OP(=O)(O[H])O[*] 0.000 claims description 4
- 238000010354 CRISPR gene editing Methods 0.000 claims description 3
- 230000000295 complement effect Effects 0.000 claims description 3
- 239000013604 expression vector Substances 0.000 claims description 3
- 239000012634 fragment Substances 0.000 claims description 3
- 102000004169 proteins and genes Human genes 0.000 claims description 3
- 238000003259 recombinant expression Methods 0.000 claims description 3
- 125000004200 2-methoxyethyl group Chemical group [H]C([H])([H])OC([H])([H])C([H])([H])* 0.000 claims description 2
- 108010042407 Endonucleases Proteins 0.000 claims description 2
- 102000004533 Endonucleases Human genes 0.000 claims description 2
- 241000233866 Fungi Species 0.000 claims description 2
- 108091027544 Subgenomic mRNA Proteins 0.000 claims description 2
- 210000001744 T-lymphocyte Anatomy 0.000 claims description 2
- 230000004913 activation Effects 0.000 claims description 2
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- 230000034431 double-strand break repair via homologous recombination Effects 0.000 claims description 2
- 238000004520 electroporation Methods 0.000 claims description 2
- 230000014509 gene expression Effects 0.000 claims description 2
- 238000010362 genome editing Methods 0.000 claims description 2
- 230000003394 haemopoietic effect Effects 0.000 claims description 2
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims description 2
- 230000001939 inductive effect Effects 0.000 claims description 2
- 230000005764 inhibitory process Effects 0.000 claims description 2
- 210000002901 mesenchymal stem cell Anatomy 0.000 claims description 2
- 210000001616 monocyte Anatomy 0.000 claims description 2
- 210000000822 natural killer cell Anatomy 0.000 claims description 2
- 210000001178 neural stem cell Anatomy 0.000 claims description 2
- 229940046166 oligodeoxynucleotide Drugs 0.000 claims description 2
- 210000000056 organ Anatomy 0.000 claims description 2
- 239000002243 precursor Substances 0.000 claims description 2
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 9
- 208000035475 disorder Diseases 0.000 description 6
- 208000026350 Inborn Genetic disease Diseases 0.000 description 3
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- 208000031886 HIV Infections Diseases 0.000 description 1
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- 208000031220 Hemophilia Diseases 0.000 description 1
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- 206010061218 Inflammation Diseases 0.000 description 1
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- 208000018737 Parkinson disease Diseases 0.000 description 1
- 102000029797 Prion Human genes 0.000 description 1
- 108091000054 Prion Proteins 0.000 description 1
- 208000012827 T-B+ severe combined immunodeficiency due to gamma chain deficiency Diseases 0.000 description 1
- 208000002903 Thalassemia Diseases 0.000 description 1
- 208000023940 X-Linked Combined Immunodeficiency disease Diseases 0.000 description 1
- 201000007146 X-linked severe combined immunodeficiency Diseases 0.000 description 1
- 206010064930 age-related macular degeneration Diseases 0.000 description 1
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 description 1
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- 208000033519 human immunodeficiency virus infectious disease Diseases 0.000 description 1
- 230000004054 inflammatory process Effects 0.000 description 1
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Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562143729P | 2015-04-06 | 2015-04-06 | |
| US62/143,729 | 2015-04-06 | ||
| US201562160545P | 2015-05-12 | 2015-05-12 | |
| US62/160,545 | 2015-05-12 | ||
| PCT/US2016/026028 WO2016164356A1 (en) | 2015-04-06 | 2016-04-05 | Chemically modified guide rnas for crispr/cas-mediated gene regulation |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018513682A JP2018513682A (ja) | 2018-05-31 |
| JP2018513682A5 true JP2018513682A5 (https=) | 2019-05-16 |
| JP6873911B2 JP6873911B2 (ja) | 2021-05-19 |
Family
ID=55910340
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017552447A Active JP6873911B2 (ja) | 2015-04-06 | 2016-04-05 | 初代細胞において標的核酸の遺伝子調節を誘導するためにインビトロで行う方法 |
Country Status (9)
| Country | Link |
|---|---|
| US (5) | US11306309B2 (https=) |
| EP (1) | EP3280803B1 (https=) |
| JP (1) | JP6873911B2 (https=) |
| KR (2) | KR102888521B1 (https=) |
| CN (2) | CN114231527A (https=) |
| AU (2) | AU2016246450B2 (https=) |
| CA (1) | CA2981715A1 (https=) |
| ES (1) | ES2884838T3 (https=) |
| WO (1) | WO2016164356A1 (https=) |
Families Citing this family (161)
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| US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
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| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
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