JP2018516556A5 - - Google Patents
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- JP2018516556A5 JP2018516556A5 JP2017559044A JP2017559044A JP2018516556A5 JP 2018516556 A5 JP2018516556 A5 JP 2018516556A5 JP 2017559044 A JP2017559044 A JP 2017559044A JP 2017559044 A JP2017559044 A JP 2017559044A JP 2018516556 A5 JP2018516556 A5 JP 2018516556A5
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- Prior art keywords
- seq
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- protein
- composition
- fusion protein
- 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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- 210000004027 cell Anatomy 0.000 claims description 63
- 108020001507 fusion proteins Proteins 0.000 claims description 20
- 102000037865 fusion proteins Human genes 0.000 claims description 20
- 102000004169 proteins and genes Human genes 0.000 claims description 20
- 108090000623 proteins and genes Proteins 0.000 claims description 20
- 238000000034 method Methods 0.000 claims description 15
- 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
- 108700028146 Genetic Enhancer Elements Proteins 0.000 claims description 12
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 10
- 101710185494 Zinc finger protein Proteins 0.000 claims description 8
- 102100023597 Zinc finger protein 816 Human genes 0.000 claims description 8
- 238000010362 genome editing Methods 0.000 claims description 8
- 210000000130 stem cell Anatomy 0.000 claims description 7
- 102100022976 B-cell lymphoma/leukemia 11A Human genes 0.000 claims description 6
- 101000903703 Homo sapiens B-cell lymphoma/leukemia 11A Proteins 0.000 claims description 6
- 238000003776 cleavage reaction Methods 0.000 claims description 4
- 238000012217 deletion Methods 0.000 claims description 4
- 230000037430 deletion Effects 0.000 claims description 4
- 208000034737 hemoglobinopathy Diseases 0.000 claims description 4
- 230000007017 scission Effects 0.000 claims description 4
- 238000013518 transcription Methods 0.000 claims description 4
- 230000035897 transcription Effects 0.000 claims description 4
- 230000002159 abnormal effect Effects 0.000 claims description 3
- 238000012986 modification Methods 0.000 claims description 3
- 230000004048 modification Effects 0.000 claims description 3
- 102000004127 Cytokines Human genes 0.000 claims description 2
- 108090000695 Cytokines Proteins 0.000 claims description 2
- 102100021519 Hemoglobin subunit beta Human genes 0.000 claims description 2
- 108091005904 Hemoglobin subunit beta Proteins 0.000 claims description 2
- 108091005886 Hemoglobin subunit gamma Proteins 0.000 claims description 2
- 102100038617 Hemoglobin subunit gamma-2 Human genes 0.000 claims description 2
- 230000004913 activation Effects 0.000 claims description 2
- 208000005980 beta thalassemia Diseases 0.000 claims description 2
- 238000010322 bone marrow transplantation Methods 0.000 claims description 2
- 102000018146 globin Human genes 0.000 claims description 2
- 108060003196 globin Proteins 0.000 claims description 2
- 210000005260 human cell Anatomy 0.000 claims description 2
- 238000003780 insertion Methods 0.000 claims description 2
- 230000037431 insertion Effects 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims description 2
- 208000007056 sickle cell anemia Diseases 0.000 claims description 2
- 230000004936 stimulating effect Effects 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 13
- 208000018337 inherited hemoglobinopathy Diseases 0.000 claims 1
- 230000035800 maturation Effects 0.000 claims 1
- 230000015572 biosynthetic process Effects 0.000 description 1
- 239000002243 precursor Substances 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021071881A JP7365374B2 (ja) | 2015-05-12 | 2021-04-21 | ヌクレアーゼ介在性遺伝子発現調節 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562160396P | 2015-05-12 | 2015-05-12 | |
| US62/160,396 | 2015-05-12 | ||
| US201662303595P | 2016-03-04 | 2016-03-04 | |
| US62/303,595 | 2016-03-04 | ||
| PCT/US2016/032049 WO2016183298A2 (en) | 2015-05-12 | 2016-05-12 | Nuclease-mediated regulation of gene expression |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021071881A Division JP7365374B2 (ja) | 2015-05-12 | 2021-04-21 | ヌクレアーゼ介在性遺伝子発現調節 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018516556A JP2018516556A (ja) | 2018-06-28 |
| JP2018516556A5 true JP2018516556A5 (enExample) | 2019-06-13 |
| JP6873917B2 JP6873917B2 (ja) | 2021-05-19 |
Family
ID=57249092
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017559044A Active JP6873917B2 (ja) | 2015-05-12 | 2016-05-12 | ヌクレアーゼ介在性遺伝子発現調節 |
| JP2021071881A Active JP7365374B2 (ja) | 2015-05-12 | 2021-04-21 | ヌクレアーゼ介在性遺伝子発現調節 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021071881A Active JP7365374B2 (ja) | 2015-05-12 | 2021-04-21 | ヌクレアーゼ介在性遺伝子発現調節 |
Country Status (17)
| Country | Link |
|---|---|
| US (2) | US10808020B2 (enExample) |
| EP (1) | EP3294866A4 (enExample) |
| JP (2) | JP6873917B2 (enExample) |
| KR (1) | KR20170141217A (enExample) |
| AU (2) | AU2016261927B2 (enExample) |
| BR (1) | BR112017024115A2 (enExample) |
| CA (1) | CA2984013A1 (enExample) |
| CL (1) | CL2017002843A1 (enExample) |
| CO (1) | CO2017012026A2 (enExample) |
| HK (1) | HK1251011A1 (enExample) |
| IL (3) | IL284707B2 (enExample) |
| MX (2) | MX382223B (enExample) |
| PH (1) | PH12017502026A1 (enExample) |
| SA (1) | SA517390307B1 (enExample) |
| SG (1) | SG10202112057QA (enExample) |
| WO (1) | WO2016183298A2 (enExample) |
| ZA (1) | ZA201707016B (enExample) |
Families Citing this family (54)
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| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| HRP20211706T1 (hr) * | 2013-11-13 | 2022-02-04 | The Children's Medical Center Corporation | Nukleazom posredovana regulacija ekspresije gena |
| US11053481B2 (en) | 2013-12-12 | 2021-07-06 | President And Fellows Of Harvard College | Fusions of Cas9 domains and nucleic acid-editing domains |
| AU2015298571B2 (en) | 2014-07-30 | 2020-09-03 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| MX382223B (es) * | 2015-05-12 | 2025-03-13 | Sangamo Therapeutics Inc | Regulacion de expresion genica mediada por nucleasa. |
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