JP2016518142A5 - - Google Patents
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- JP2016518142A5 JP2016518142A5 JP2016513016A JP2016513016A JP2016518142A5 JP 2016518142 A5 JP2016518142 A5 JP 2016518142A5 JP 2016513016 A JP2016513016 A JP 2016513016A JP 2016513016 A JP2016513016 A JP 2016513016A JP 2016518142 A5 JP2016518142 A5 JP 2016518142A5
- Authority
- JP
- Japan
- Prior art keywords
- nuclease
- dna
- combination
- sequence
- composition
- 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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- 210000004027 cell Anatomy 0.000 claims description 49
- 101710163270 Nuclease Proteins 0.000 claims description 37
- 108020004414 DNA Proteins 0.000 claims description 35
- 108090000623 proteins and genes Proteins 0.000 claims description 34
- 108091033319 polynucleotide Proteins 0.000 claims description 14
- 102000040430 polynucleotide Human genes 0.000 claims description 14
- 239000002157 polynucleotide Substances 0.000 claims description 14
- 230000010354 integration Effects 0.000 claims description 9
- 231100000331 toxic Toxicity 0.000 claims description 8
- 230000002588 toxic effect Effects 0.000 claims description 8
- 238000003776 cleavage reaction Methods 0.000 claims description 7
- 230000007017 scission Effects 0.000 claims description 7
- 108091026890 Coding region Proteins 0.000 claims description 6
- 239000013600 plasmid vector Substances 0.000 claims description 6
- 102000004169 proteins and genes Human genes 0.000 claims description 6
- 210000000130 stem cell Anatomy 0.000 claims description 6
- 108700019146 Transgenes Proteins 0.000 claims description 5
- 108010017070 Zinc Finger Nucleases Proteins 0.000 claims description 5
- 239000013603 viral vector Substances 0.000 claims description 5
- 108091030071 RNAI Proteins 0.000 claims description 4
- 108091027967 Small hairpin RNA Proteins 0.000 claims description 4
- 230000009368 gene silencing by RNA Effects 0.000 claims description 4
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims description 4
- -1 lymphokine Proteins 0.000 claims description 4
- 230000001404 mediated effect Effects 0.000 claims description 4
- 108091070501 miRNA Proteins 0.000 claims description 4
- 239000002679 microRNA Substances 0.000 claims description 4
- 238000012986 modification Methods 0.000 claims description 4
- 230000004048 modification Effects 0.000 claims description 4
- 239000004055 small Interfering RNA Substances 0.000 claims description 4
- 108091033409 CRISPR Proteins 0.000 claims description 3
- 238000010354 CRISPR gene editing Methods 0.000 claims description 3
- 108010001857 Cell Surface Receptors Proteins 0.000 claims description 2
- 102000004127 Cytokines Human genes 0.000 claims description 2
- 108090000695 Cytokines Proteins 0.000 claims description 2
- 108090000790 Enzymes Proteins 0.000 claims description 2
- 102000004190 Enzymes Human genes 0.000 claims description 2
- 102000007399 Nuclear hormone receptor Human genes 0.000 claims description 2
- 108020005497 Nuclear hormone receptor Proteins 0.000 claims description 2
- 108010073062 Transcription Activator-Like Effectors Proteins 0.000 claims description 2
- 239000000427 antigen Substances 0.000 claims description 2
- 108091007433 antigens Proteins 0.000 claims description 2
- 102000036639 antigens Human genes 0.000 claims description 2
- 239000003153 chemical reaction reagent Substances 0.000 claims description 2
- 239000003102 growth factor Substances 0.000 claims description 2
- 229940088597 hormone Drugs 0.000 claims description 2
- 239000005556 hormone Substances 0.000 claims description 2
- 210000004962 mammalian cell Anatomy 0.000 claims description 2
- 102000006240 membrane receptors Human genes 0.000 claims description 2
- 108020004999 messenger RNA Proteins 0.000 claims description 2
- 230000001400 myeloablative effect Effects 0.000 claims description 2
- 108020004017 nuclear receptors Proteins 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 27
- 101800004910 Nuclease A Proteins 0.000 claims 2
- 101150090724 3 gene Proteins 0.000 claims 1
- 210000001185 bone marrow Anatomy 0.000 claims 1
- 230000001419 dependent effect Effects 0.000 claims 1
- 238000000034 method Methods 0.000 description 22
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 1
- 241000700605 Viruses Species 0.000 description 1
- 238000010322 bone marrow transplantation Methods 0.000 description 1
- 239000013604 expression vector Substances 0.000 description 1
- 230000009261 transgenic effect Effects 0.000 description 1
- 239000013598 vector Substances 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361821872P | 2013-05-10 | 2013-05-10 | |
| US61/821,872 | 2013-05-10 | ||
| PCT/US2014/036972 WO2014182700A1 (en) | 2013-05-10 | 2014-05-06 | Delivery methods and compositions for nuclease-mediated genome engineering |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2016518142A JP2016518142A (ja) | 2016-06-23 |
| JP2016518142A5 true JP2016518142A5 (https=) | 2017-03-23 |
Family
ID=51864932
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016513016A Pending JP2016518142A (ja) | 2013-05-10 | 2014-05-06 | ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法および組成物 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US10604771B2 (https=) |
| EP (1) | EP2994531B1 (https=) |
| JP (1) | JP2016518142A (https=) |
| AU (1) | AU2014262867B2 (https=) |
| CA (1) | CA2910427C (https=) |
| WO (1) | WO2014182700A1 (https=) |
Families Citing this family (122)
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| JP6261500B2 (ja) | 2011-07-22 | 2018-01-17 | プレジデント アンド フェローズ オブ ハーバード カレッジ | ヌクレアーゼ切断特異性の評価および改善 |
| EP3241902B1 (en) | 2012-05-25 | 2018-02-28 | The Regents of The University of California | Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription |
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| AU2014227653B2 (en) | 2013-03-15 | 2017-04-20 | The General Hospital Corporation | Using RNA-guided foki nucleases (RFNs) to increase specificity for RNA-guided genome editing |
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- 2014-05-06 AU AU2014262867A patent/AU2014262867B2/en not_active Ceased
- 2014-05-06 EP EP14794094.4A patent/EP2994531B1/en active Active
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