Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice.
Nat Commun. 2021 Jan 29;12(1):686. doi: 10.1038/s41467-021-20909-x.
Nat Commun. 2021.
PMID: 33514718
Free PMC article.