JP2017532955A5 - - Google Patents
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- JP2017532955A5 JP2017532955A5 JP2017514521A JP2017514521A JP2017532955A5 JP 2017532955 A5 JP2017532955 A5 JP 2017532955A5 JP 2017514521 A JP2017514521 A JP 2017514521A JP 2017514521 A JP2017514521 A JP 2017514521A JP 2017532955 A5 JP2017532955 A5 JP 2017532955A5
- Authority
- JP
- Japan
- Prior art keywords
- cell
- genetically modified
- zinc finger
- modified cell
- item
- 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 56
- 101150013707 HBB gene Proteins 0.000 claims description 16
- 238000000034 method Methods 0.000 claims description 16
- 108091033319 polynucleotide Proteins 0.000 claims description 16
- 102000040430 polynucleotide Human genes 0.000 claims description 16
- 239000002157 polynucleotide Substances 0.000 claims description 16
- 210000003743 erythrocyte Anatomy 0.000 claims description 12
- 108020001507 fusion proteins Proteins 0.000 claims description 10
- 102000037865 fusion proteins Human genes 0.000 claims description 10
- 230000014509 gene expression Effects 0.000 claims description 10
- 108090000623 proteins and genes Proteins 0.000 claims description 10
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims description 8
- 102000004169 proteins and genes Human genes 0.000 claims description 8
- 101710163270 Nuclease Proteins 0.000 claims description 6
- 108010017070 Zinc Finger Nucleases Proteins 0.000 claims description 6
- 101710185494 Zinc finger protein Proteins 0.000 claims description 6
- 102100023597 Zinc finger protein 816 Human genes 0.000 claims description 6
- 238000010362 genome editing Methods 0.000 claims description 6
- 210000000130 stem cell Anatomy 0.000 claims description 6
- 239000008194 pharmaceutical composition Substances 0.000 claims description 5
- HCHKCACWOHOZIP-UHFFFAOYSA-N Zinc Chemical compound [Zn] HCHKCACWOHOZIP-UHFFFAOYSA-N 0.000 claims description 4
- 238000003780 insertion Methods 0.000 claims description 4
- 230000037431 insertion Effects 0.000 claims description 4
- 238000004519 manufacturing process Methods 0.000 claims description 4
- 230000035772 mutation Effects 0.000 claims description 4
- 239000002243 precursor Substances 0.000 claims description 4
- 239000011701 zinc Substances 0.000 claims description 4
- 229910052725 zinc Inorganic materials 0.000 claims description 4
- 102000004127 Cytokines Human genes 0.000 claims description 2
- 108090000695 Cytokines Proteins 0.000 claims description 2
- 102100031573 Hematopoietic progenitor cell antigen CD34 Human genes 0.000 claims description 2
- 101000777663 Homo sapiens Hematopoietic progenitor cell antigen CD34 Proteins 0.000 claims description 2
- 108091034117 Oligonucleotide Proteins 0.000 claims description 2
- 108020005067 RNA Splice Sites Proteins 0.000 claims description 2
- 108700019146 Transgenes Proteins 0.000 claims description 2
- 208000005980 beta thalassemia Diseases 0.000 claims description 2
- 238000003776 cleavage reaction Methods 0.000 claims description 2
- 238000012217 deletion Methods 0.000 claims description 2
- 230000037430 deletion Effects 0.000 claims description 2
- 238000012239 gene modification Methods 0.000 claims description 2
- 230000005017 genetic modification Effects 0.000 claims description 2
- 235000013617 genetically modified food Nutrition 0.000 claims description 2
- 238000010348 incorporation Methods 0.000 claims description 2
- 108020004999 messenger RNA Proteins 0.000 claims description 2
- 238000012545 processing Methods 0.000 claims description 2
- 230000007017 scission Effects 0.000 claims description 2
- 230000004936 stimulating effect Effects 0.000 claims description 2
- 238000000338 in vitro Methods 0.000 claims 2
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2019150566A JP6929585B2 (ja) | 2014-09-16 | 2019-08-20 | 造血幹細胞におけるヌクレアーゼ媒介性ゲノム工学および補正のための方法および組成物 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462051159P | 2014-09-16 | 2014-09-16 | |
| US62/051,159 | 2014-09-16 | ||
| PCT/US2015/050411 WO2016044416A1 (en) | 2014-09-16 | 2015-09-16 | Methods and compositions for nuclease-mediated genome engineering and correction in hematopoietic stem cells |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019150566A Division JP6929585B2 (ja) | 2014-09-16 | 2019-08-20 | 造血幹細胞におけるヌクレアーゼ媒介性ゲノム工学および補正のための方法および組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017532955A JP2017532955A (ja) | 2017-11-09 |
| JP2017532955A5 true JP2017532955A5 (OSRAM) | 2018-08-16 |
| JP6722176B2 JP6722176B2 (ja) | 2020-07-15 |
Family
ID=55533790
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017514521A Expired - Fee Related JP6722176B2 (ja) | 2014-09-16 | 2015-09-16 | 造血幹細胞におけるヌクレアーゼ媒介性ゲノム工学および補正のための方法および組成物 |
| JP2019150566A Expired - Fee Related JP6929585B2 (ja) | 2014-09-16 | 2019-08-20 | 造血幹細胞におけるヌクレアーゼ媒介性ゲノム工学および補正のための方法および組成物 |
| JP2021128240A Pending JP2021182931A (ja) | 2014-09-16 | 2021-08-04 | 造血幹細胞におけるヌクレアーゼ媒介性ゲノム工学および補正のための方法および組成物 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019150566A Expired - Fee Related JP6929585B2 (ja) | 2014-09-16 | 2019-08-20 | 造血幹細胞におけるヌクレアーゼ媒介性ゲノム工学および補正のための方法および組成物 |
| JP2021128240A Pending JP2021182931A (ja) | 2014-09-16 | 2021-08-04 | 造血幹細胞におけるヌクレアーゼ媒介性ゲノム工学および補正のための方法および組成物 |
Country Status (18)
| Country | Link |
|---|---|
| US (1) | US10435677B2 (OSRAM) |
| EP (2) | EP3878948A1 (OSRAM) |
| JP (3) | JP6722176B2 (OSRAM) |
| KR (1) | KR20170054445A (OSRAM) |
| CN (2) | CN106795488B (OSRAM) |
| AU (2) | AU2015317793B2 (OSRAM) |
| CA (1) | CA2960769A1 (OSRAM) |
| DK (1) | DK3194570T3 (OSRAM) |
| ES (1) | ES2886012T3 (OSRAM) |
| HR (1) | HRP20211468T1 (OSRAM) |
| HU (1) | HUE055583T2 (OSRAM) |
| LT (1) | LT3194570T (OSRAM) |
| PL (1) | PL3194570T3 (OSRAM) |
| PT (1) | PT3194570T (OSRAM) |
| RS (1) | RS62334B1 (OSRAM) |
| SG (2) | SG10202011572XA (OSRAM) |
| SI (1) | SI3194570T1 (OSRAM) |
| WO (1) | WO2016044416A1 (OSRAM) |
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| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| 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 |
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