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Reactivation of γ-globin in adult β-YAC mice after ex vivo and in vivo hematopoietic stem cell genome editing
| Content Provider | Scilit |
|---|---|
| Author | Li, Chang Psatha, Nikoletta Sova, Pavel Gil, Sucheol Wang, Hongjie Kim, Jiho Kulkarni, Chandana Valensisi, Cristina Hawkins, R. David Stamatoyannopoulos, George Lieber, André |
| Copyright Year | 2018 |
| Description | Key Points CRISPR/Cas9-mediated disruption of a BCL11A binding site in HSCs of β-YAC mice results in the reactivation of γ-globin in erythrocytes. Our approach for in vivo HSC genome editing that does not require HSC transplantation and myeloablation should simplify HSC gene therapy. |
| Related Links | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6024639/pdf |
| Ending Page | 2928 |
| Page Count | 14 |
| Starting Page | 2915 |
| DOI | 10.1182/blood-2018-03-838540 |
| Journal | Blood |
| Issue Number | 26 |
| Volume Number | 131 |
| Language | English |
| Publisher | American Society of Hematology |
| Publisher Date | 2018-06-28 |
| Access Restriction | Open |
| Subject Keyword | Research and Experimental Medicine Hematopoietic Gene Therapy Transplantation Adult Yac Globin Simplify Myeloablation Journal: Blood (Vol- 2019, Issue- 26) |
| Content Type | Text |
| Resource Type | Article |