Article
Genetic correction of concurrent α- and β-thalassemia patient-derived pluripotent stem cells by the CRISPR-Cas9 technology.
Stem cell research & therapy - 7 Mar 2022
Li Lingli, Yi Hongyan, Liu Zheng, Long Ping, Pan Tao, Huang Yuanhua, Li Yongsheng, Li Qi, Ma Yanlin
Abstract excerpt
BACKGROUND: Thalassemia is a genetic blood disorder characterized by decreased hemoglobin production. Severe anemia can damage organs and severe threat to life safety. Allogeneic transplantation of bone marrow-derived hematopoietic stem cell (HSCs) at present represents a promising therapeutic approach for thalassemia. However, immune rejection and lack of HLA-matched donors limited its clinical application. In...
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