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

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

Abstract 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 therapeut...

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Bibliographic Details
Main Authors: Lingli Li, Hongyan Yi, Zheng Liu, Ping Long, Tao Pan, Yuanhua Huang, Yongsheng Li, Qi Li, Yanlin Ma
Format: Article
Language:English
Published: BMC 2022-03-01
Series:Stem Cell Research & Therapy
Subjects:
Thalassemia
CRISPR-Cas9 technology
Human-induced pluripotent stem cells
HBB gene
HBA2 gene
Online Access:https://doi.org/10.1186/s13287-022-02768-5

Internet

https://doi.org/10.1186/s13287-022-02768-5

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