JP2017500061A5 - - Google Patents
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- JP2017500061A5 JP2017500061A5 JP2016557545A JP2016557545A JP2017500061A5 JP 2017500061 A5 JP2017500061 A5 JP 2017500061A5 JP 2016557545 A JP2016557545 A JP 2016557545A JP 2016557545 A JP2016557545 A JP 2016557545A JP 2017500061 A5 JP2017500061 A5 JP 2017500061A5
- Authority
- JP
- Japan
- Prior art keywords
- zinc finger
- composition
- expression vector
- protein
- vector
- 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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- 239000013604 expression vector Substances 0.000 claims description 17
- 102000040430 polynucleotide Human genes 0.000 claims description 12
- 108091033319 polynucleotide Proteins 0.000 claims description 12
- 239000002157 polynucleotide Substances 0.000 claims description 12
- 101710185494 Zinc finger protein Proteins 0.000 claims description 11
- 102100023597 Zinc finger protein 816 Human genes 0.000 claims description 11
- 208000015439 Lysosomal storage disease Diseases 0.000 claims description 10
- HCHKCACWOHOZIP-UHFFFAOYSA-N Zinc Chemical compound [Zn] HCHKCACWOHOZIP-UHFFFAOYSA-N 0.000 claims description 10
- 239000008194 pharmaceutical composition Substances 0.000 claims description 10
- 108090000623 proteins and genes Proteins 0.000 claims description 10
- 102000004169 proteins and genes Human genes 0.000 claims description 10
- 239000011701 zinc Substances 0.000 claims description 10
- 229910052725 zinc Inorganic materials 0.000 claims description 10
- 239000013607 AAV vector Substances 0.000 claims description 8
- 108010088751 Albumins Proteins 0.000 claims description 8
- 108010017070 Zinc Finger Nucleases Proteins 0.000 claims description 8
- 238000003776 cleavage reaction Methods 0.000 claims description 8
- 230000007017 scission Effects 0.000 claims description 8
- 210000004027 cell Anatomy 0.000 claims description 6
- 239000013598 vector Substances 0.000 claims description 6
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 4
- 108700019146 Transgenes Proteins 0.000 claims description 4
- 230000002950 deficient Effects 0.000 claims description 2
- 108020001507 fusion proteins Proteins 0.000 claims description 2
- 102000037865 fusion proteins Human genes 0.000 claims description 2
- 230000010354 integration Effects 0.000 claims description 2
- 210000005229 liver cell Anatomy 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 15
- 238000000034 method Methods 0.000 description 11
- 238000010348 incorporation Methods 0.000 description 1
- 238000012986 modification Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361913838P | 2013-12-09 | 2013-12-09 | |
| US61/913,838 | 2013-12-09 | ||
| US201461943884P | 2014-02-24 | 2014-02-24 | |
| US61/943,884 | 2014-02-24 | ||
| PCT/US2014/069352 WO2015089077A2 (en) | 2013-12-09 | 2014-12-09 | Methods and compositions for genome engineering |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018236131A Division JP2019037254A (ja) | 2013-12-09 | 2018-12-18 | ゲノム操作のための方法および組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017500061A JP2017500061A (ja) | 2017-01-05 |
| JP2017500061A5 true JP2017500061A5 (enExample) | 2017-12-21 |
| JP6535684B2 JP6535684B2 (ja) | 2019-06-26 |
Family
ID=53270543
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016557545A Expired - Fee Related JP6535684B2 (ja) | 2013-12-09 | 2014-12-09 | ゲノム操作のための方法および組成物 |
| JP2016557544A Expired - Fee Related JP6642943B2 (ja) | 2013-12-09 | 2014-12-09 | 血友病を処置するための方法および組成物 |
| JP2018236131A Pending JP2019037254A (ja) | 2013-12-09 | 2018-12-18 | ゲノム操作のための方法および組成物 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016557544A Expired - Fee Related JP6642943B2 (ja) | 2013-12-09 | 2014-12-09 | 血友病を処置するための方法および組成物 |
| JP2018236131A Pending JP2019037254A (ja) | 2013-12-09 | 2018-12-18 | ゲノム操作のための方法および組成物 |
Country Status (11)
| Country | Link |
|---|---|
| US (6) | US10081661B2 (enExample) |
| EP (3) | EP3080143B1 (enExample) |
| JP (3) | JP6535684B2 (enExample) |
| CN (3) | CN111330021A (enExample) |
| AU (3) | AU2014364051B2 (enExample) |
| CA (3) | CA3229275A1 (enExample) |
| DK (1) | DK3080274T3 (enExample) |
| ES (1) | ES2813367T3 (enExample) |
| HU (1) | HUE051628T2 (enExample) |
| IL (3) | IL245785B (enExample) |
| WO (2) | WO2015089077A2 (enExample) |
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