Genetic correction of concurrent α- and β-thalassemia patient-derived pluripotent stem cells by the CRISPR-Cas9 technology
Summary
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 |