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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...
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| Gepubliceerd in: | Blood Adv |
|---|---|
| Hoofdauteurs: | , , , , , , |
| Formaat: | Artigo |
| Taal: | Inglês |
| Gepubliceerd in: |
American Society of Hematology
2021
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| Onderwerpen: | |
| Online toegang: | 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 |
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