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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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Bibliografische gegevens
Gepubliceerd in:Blood Adv
Hoofdauteurs: Li, Chang, Georgakopoulou, Aphrodite, Mishra, Arpit, Gil, Sucheol, Hawkins, R. David, Yannaki, Evangelia, Lieber, André
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: American Society of Hematology 2021
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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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