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

Humbert O, Radtke S, Samuelson C, Carrillo RR, Perez AM, Reddy SS, Lux C, Pattabhi S, Schefter LE, Negre O, Lee CM, Bao G, Adair JE, Peterson CW, Rawlings DJ, Scharenberg AM, Kiem HP. Therapeutically relevant engraftment of a CRISPR-Cas9-edited HSC-enriched population with HbF reactivation in nonhuman primates. Sci Transl Med. 2019 07 31; 11(503).

View in: PubMed