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
PubMed 35255977
PubMed Central PMC8900422
DOI 10.1186/s13287-022-02768-5

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