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Journal Articles

In vivo HSPC gene therapy with base editors allows for efficient reactivation of fetal γ-globin in β-YAC mice

Chang Li, Aphrodite Georgakopoulou, Arpit Mishra, Sucheol Gil, R. David Hawkins, Evangelia Yannaki, André Lieber
Journal: Blood Advances
Blood Adv (2021) 5 (4): 1122–1135.
https://doi.org/10.1182/bloodadvances.2020003702
Published: 2021
Includes: Supplemental data