The safety and effectiveness of genetically corrected iPSCs derived from β-thalassaemia patients in nonmyeloablative β-thalassaemic mice
Summary
Our study provides evidence that the transplantation of genetically corrected, patient-specific iPSCs could be used to cure genetic diseases, such as β-thalassaemia major.
| Authors | Xian Y, Xie Y, Song B, Ou Z, Ouyang S, Xie Y, Yang Y, Xiong Z, Li H, Sun X |
|---|---|
| Journal | Stem cell research & therapy |
| Publication Date | 2020 Jul 16;11(1):288 |
| PubMed | 32678022 |
| PubMed Central | PMC7367314 |
| DOI | 10.1186/s13287-020-01765-w |