JP2017535271A5 - - Google Patents
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- JP2017535271A5 JP2017535271A5 JP2017527267A JP2017527267A JP2017535271A5 JP 2017535271 A5 JP2017535271 A5 JP 2017535271A5 JP 2017527267 A JP2017527267 A JP 2017527267A JP 2017527267 A JP2017527267 A JP 2017527267A JP 2017535271 A5 JP2017535271 A5 JP 2017535271A5
- Authority
- JP
- Japan
- Prior art keywords
- cell
- crispr rna
- sequence
- rna recognition
- farther
- 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.)
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- 108091079001 CRISPR RNA Proteins 0.000 claims description 213
- 238000000034 method Methods 0.000 claims description 161
- 210000004027 cell Anatomy 0.000 claims description 159
- 108090000623 proteins and genes Proteins 0.000 claims description 120
- 102000004169 proteins and genes Human genes 0.000 claims description 84
- 108020004707 nucleic acids Proteins 0.000 claims description 83
- 102000039446 nucleic acids Human genes 0.000 claims description 83
- 150000007523 nucleic acids Chemical class 0.000 claims description 83
- 239000000523 sample Substances 0.000 claims description 82
- 210000000349 chromosome Anatomy 0.000 claims description 78
- 108700028369 Alleles Proteins 0.000 claims description 76
- 108020004414 DNA Proteins 0.000 claims description 74
- 108020005004 Guide RNA Proteins 0.000 claims description 56
- 230000008685 targeting Effects 0.000 claims description 39
- 108091028113 Trans-activating crRNA Proteins 0.000 claims description 29
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 25
- 230000005782 double-strand break Effects 0.000 claims description 24
- 230000004048 modification Effects 0.000 claims description 24
- 238000012986 modification Methods 0.000 claims description 24
- 230000037431 insertion Effects 0.000 claims description 23
- 238000003780 insertion Methods 0.000 claims description 23
- 230000037430 deletion Effects 0.000 claims description 22
- 238000012217 deletion Methods 0.000 claims description 22
- 210000001161 mammalian embryo Anatomy 0.000 claims description 22
- 239000002773 nucleotide Substances 0.000 claims description 22
- 125000003729 nucleotide group Chemical group 0.000 claims description 22
- 230000006780 non-homologous end joining Effects 0.000 claims description 13
- 230000000694 effects Effects 0.000 claims description 9
- 230000035772 mutation Effects 0.000 claims description 9
- 241000283984 Rodentia Species 0.000 claims description 8
- 210000003527 eukaryotic cell Anatomy 0.000 claims description 8
- 210000005260 human cell Anatomy 0.000 claims description 8
- 108020004999 messenger RNA Proteins 0.000 claims description 6
- 108091033409 CRISPR Proteins 0.000 claims description 5
- 102000053602 DNA Human genes 0.000 claims description 5
- 108010008532 Deoxyribonuclease I Proteins 0.000 claims description 5
- 102000007260 Deoxyribonuclease I Human genes 0.000 claims description 5
- 238000006243 chemical reaction Methods 0.000 claims description 5
- 230000006798 recombination Effects 0.000 claims description 5
- 238000005215 recombination Methods 0.000 claims description 5
- 210000004504 adult stem cell Anatomy 0.000 claims description 4
- 230000003834 intracellular effect Effects 0.000 claims description 4
- 210000004962 mammalian cell Anatomy 0.000 claims description 4
- 210000000130 stem cell Anatomy 0.000 claims description 4
- 102000003960 Ligases Human genes 0.000 claims description 3
- 108090000364 Ligases Proteins 0.000 claims description 3
- 101710163270 Nuclease Proteins 0.000 claims description 3
- 108010064218 Poly (ADP-Ribose) Polymerase-1 Proteins 0.000 claims description 3
- 102000015087 Poly (ADP-Ribose) Polymerase-1 Human genes 0.000 claims description 3
- 230000004075 alteration Effects 0.000 claims description 3
- 230000034431 double-strand break repair via homologous recombination Effects 0.000 claims description 3
- 230000001939 inductive effect Effects 0.000 claims description 3
- 230000002441 reversible effect Effects 0.000 claims description 3
- 206010059866 Drug resistance Diseases 0.000 claims description 2
- 108020004682 Single-Stranded DNA Proteins 0.000 claims description 2
- 210000002230 centromere Anatomy 0.000 claims description 2
- 210000001840 diploid cell Anatomy 0.000 claims description 2
- 201000010099 disease Diseases 0.000 claims description 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 2
- 238000003556 assay Methods 0.000 claims 18
- 230000014759 maintenance of location Effects 0.000 claims 6
- 230000001404 mediated effect Effects 0.000 claims 4
- 230000000717 retained effect Effects 0.000 claims 4
- 230000003247 decreasing effect Effects 0.000 claims 2
- 238000000520 microinjection Methods 0.000 claims 2
- 108091026890 Coding region Proteins 0.000 claims 1
- 210000000805 cytoplasm Anatomy 0.000 claims 1
- 230000006378 damage Effects 0.000 claims 1
- 238000010354 CRISPR gene editing Methods 0.000 description 2
- 241000282412 Homo Species 0.000 description 2
- 238000004519 manufacturing process Methods 0.000 description 1
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462083005P | 2014-11-21 | 2014-11-21 | |
| US62/083,005 | 2014-11-21 | ||
| US201562182314P | 2015-06-19 | 2015-06-19 | |
| US62/182,314 | 2015-06-19 | ||
| US201562211421P | 2015-08-28 | 2015-08-28 | |
| US62/211,421 | 2015-08-28 | ||
| PCT/US2015/062023 WO2016081923A2 (en) | 2014-11-21 | 2015-11-20 | METHODS AND COMPOSITIONS FOR TARGETED GENETIC MODIFICATION USING PAIRED GUIDE RNAs |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020112438A Division JP7101211B2 (ja) | 2014-11-21 | 2020-06-30 | ガイドｒｎａのペアを使用したターゲティングによる遺伝子改変の方法及び組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017535271A JP2017535271A (ja) | 2017-11-30 |
| JP2017535271A5 true JP2017535271A5 (enExample) | 2018-12-20 |
| JP6727199B2 JP6727199B2 (ja) | 2020-07-22 |
Family
ID=54771219
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017527267A Active JP6727199B2 (ja) | 2014-11-21 | 2015-11-20 | ガイドｒｎａのペアを使用したターゲティングによる遺伝子改変の方法及び組成物 |
| JP2020112438A Active JP7101211B2 (ja) | 2014-11-21 | 2020-06-30 | ガイドｒｎａのペアを使用したターゲティングによる遺伝子改変の方法及び組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020112438A Active JP7101211B2 (ja) | 2014-11-21 | 2020-06-30 | ガイドｒｎａのペアを使用したターゲティングによる遺伝子改変の方法及び組成物 |
Country Status (25)
| Country | Link |
|---|---|
| US (4) | US10457960B2 (enExample) |
| EP (2) | EP3221457B1 (enExample) |
| JP (2) | JP6727199B2 (enExample) |
| KR (3) | KR102415093B1 (enExample) |
| CN (2) | CN113444747B (enExample) |
| AU (2) | AU2015349692B2 (enExample) |
| BR (1) | BR112017010547A2 (enExample) |
| CA (2) | CA3176380A1 (enExample) |
| CY (1) | CY1121738T1 (enExample) |
| DK (1) | DK3221457T3 (enExample) |
| ES (2) | ES2731437T3 (enExample) |
| HR (1) | HRP20190949T1 (enExample) |
| HU (1) | HUE044907T2 (enExample) |
| IL (3) | IL301900A (enExample) |
| LT (1) | LT3221457T (enExample) |
| MX (2) | MX389266B (enExample) |
| NZ (1) | NZ731962A (enExample) |
| PL (1) | PL3221457T3 (enExample) |
| PT (1) | PT3221457T (enExample) |
| RS (1) | RS58893B1 (enExample) |
| RU (2) | RU2020134412A (enExample) |
| SG (2) | SG11201703747RA (enExample) |
| SI (1) | SI3221457T1 (enExample) |
| SM (1) | SMT201900354T1 (enExample) |
| WO (1) | WO2016081923A2 (enExample) |
Families Citing this family (142)
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| US6596541B2 (en) | 2000-10-31 | 2003-07-22 | Regeneron Pharmaceuticals, Inc. | Methods of modifying eukaryotic cells |
| US20050144655A1 (en) | 2000-10-31 | 2005-06-30 | Economides Aris N. | Methods of modifying eukaryotic cells |
| US20100069614A1 (en) | 2008-06-27 | 2010-03-18 | Merus B.V. | Antibody producing non-human mammals |
| WO2004106375A1 (en) | 2003-05-30 | 2004-12-09 | Merus Biopharmaceuticals B.V. I.O. | Fab library for the preparation of anti vegf and anti rabies virus fabs |
| WO2013066438A2 (en) | 2011-07-22 | 2013-05-10 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| EP2841572B1 (en) | 2012-04-27 | 2019-06-19 | Duke University | Genetic correction of mutated genes |
| MX2015010841A (es) | 2013-02-20 | 2016-05-09 | Regeneron Pharma | Modificacion genetica de ratas. |
| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| DK3456831T3 (da) | 2013-04-16 | 2021-09-06 | Regeneron Pharma | Målrettet modifikation af rottegenom |
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| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
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| DK3152312T3 (da) | 2014-06-06 | 2020-04-06 | Regeneron Pharma | Fremgangsmåder og sammensætninger til modifikation af et mållocus |
| DK3161128T3 (en) | 2014-06-26 | 2018-11-05 | Regeneron Pharma | METHODS AND COMPOSITIONS FOR TARGETED GENTICAL MODIFICATIONS AND PROCEDURES FOR USE THEREOF |
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| BR112017007770A2 (pt) | 2014-10-15 | 2018-01-16 | Regeneron Pharma | cultura in vitro, população de hipscs, método para modificar um locus-alvo genômico, e, hipsc. |
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