Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease.
Lattanzi A, Camarena J, Lahiri P, Segal H, Srifa W, Vakulskas CA, Frock RL, Kenrick J, Lee C, Talbott N, Skowronski J, Cromer MK, Charlesworth CT, Bak RO, Mantri S, Bao G, DiGiusto D, Tisdale J, Wright JF, Bhatia N, Roncarolo MG, Dever DP, Porteus MH.
Lattanzi A, et al.
Sci Transl Med. 2021 Jun 16;13(598):eabf2444. doi: 10.1126/scitranslmed.abf2444.
Sci Transl Med. 2021.
PMID: 34135108
Free PMC article.