Correction of beta-thalassemia by CRISPR/Cas9 editing of the alpha-globin locus in human hematopoietic stem cells.
Pavani G, Fabiano A, Laurent M, Amor F, Cantelli E, Chalumeau A, Maule G, Tachtsidi A, Concordet JP, Cereseto A, Mavilio F, Ferrari G, Miccio A, Amendola M.
Pavani G, et al.
Blood Adv. 2021 Mar 9;5(5):1137-1153. doi: 10.1182/bloodadvances.2020001996.
Blood Adv. 2021.
PMID: 33635334
Free PMC article.
Clinical data show that severity of beta-thal correlates with the number of inherited alpha-globin genes (HBA1 and HBA2), with alpha-globin gene deletions having a beneficial effect for patients. Here, we describe a novel strategy to treat beta-thal based on genome editing …
Clinical data show that severity of beta-thal correlates with the number of inherited alpha-globin genes (HBA1 and HBA2), with alpha- …