| 发明名称 |
THERAPEUTIC USES OF GENOME EDITING WITH CRISPR/Cas SYSTEMS |
| 摘要 |
Disclosed herein are methods, compositions, and kits for high efficiency, site-specific genomic editing of cells for treating or preventing genetic blood disorders. |
| 申请公布号 |
US2015152436(A1) |
申请公布日期 |
2015.06.04 |
| 申请号 |
US201414509924 |
申请日期 |
2014.10.08 |
| 申请人 |
President and Fellows of Harvard College ;Children's Medical Center Corporation |
发明人 |
Musunuru Kiran;Cowan Chad A.;Rossi Derrick J. |
| 分类号 |
C12N15/90;A61K38/46 |
主分类号 |
C12N15/90 |
| 代理机构 |
|
代理人 |
|
| 主权项 |
1. A method for altering a target severe combined immunodeficiency (SCID)-associated polynucleotide sequence in a cell comprising contacting the SCID-associated polynucleotide sequence with a clustered regularly interspaced short palindromic repeats-associated (Cas) protein and from one to two ribonucleic acids, wherein the ribonucleic acids direct Cas protein to and hybridize to a target motif of the target SCID-associated polynucleotide sequence, wherein the target SCID-associated polynucleotide sequence is cleaved. |
| 地址 |
Cambridge MA US |
