Genetic correction of concurrent α- and β-thalassemia patient-derived pluripotent stem cells by the CRISPR-Cas9 technology
We designed sgRNAs and demonstrated that these sgRNAs facilitating the CRISPR-Cas9 genomic editing system could be applied to correct concurrent α- and β-thalassemia in patient-derived hiPSCs. In the future, these corrected hiPSCs can be applied for autologous transplantation in patients with concurrent α- and β-thalassemia. © 2022. The Author(s).
|Authors||Li L, Yi H, Liu Z, Long P, Pan T, Huang Y, Li Y, Li Q, Ma Y|
|Journal||Stem cell research & therapy|
|Publication Date||2022 Mar 7;13(1):102|