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.
Humbert O, et al.
Sci Transl Med. 2019 Jul 31;11(503):eaaw3768. doi: 10.1126/scitranslmed.aaw3768.
Sci Transl Med. 2019.
PMID: 31366580
Free PMC article.