The safety and effectiveness of genetically corrected iPSCs derived from β-thalassaemia patients in nonmyeloablative β-thalassaemic mice


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

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