載入...
In vivo HSPC gene therapy with base editors allows for efficient reactivation of fetal γ-globin in β-YAC mice
Base editors are capable of installing precise genomic alterations without creating double-strand DNA breaks. In this study, we targeted critical motifs regulating γ-globin reactivation with base editors delivered via HDAd5/35(++) vectors. Through optimized design, we successfully produced a panel o...
Na minha lista:
| 發表在: | Blood Adv |
|---|---|
| Main Authors: | , , , , , , |
| 格式: | Artigo |
| 語言: | Inglês |
| 出版: |
American Society of Hematology
2021
|
| 主題: | |
| 在線閱讀: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7903237/ https://ncbi.nlm.nih.gov/pubmed/33620431 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/bloodadvances.2020003702 |
| 標簽: |
添加標簽
沒有標簽, 成為第一個標記此記錄!
|