Therapeutically relevant engraftment of a CRISPR-Cas9-edited HSC-enriched population with HbF reactivation in nonhuman primates

Humbert, Olivier ; Radtke, Stefan ; Samuelson, Clare ; Carrillo, Ray R ; Perez, Anai M ; Reddy, Sowmya S ; Lux, Christopher ; Pattabhi, Sowmya ; Schefter, Lauren E ; Negre, Olivier ; Lee, Ciaran M ; Bao, Gang ; Adair, Jennifer E ; Peterson, Christopher W ; Rawlings, David J ; Scharenberg, Andrew M ; Kiem, Hans-Peter

Science translational medicine, 2019-07, Vol.11 (503) [Periódico revisado por pares]

United States: The American Association for the Advancement of Science

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