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

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 appro...

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Detalles Bibliográficos
Autores principales: Li, Lingli, Yi, Hongyan, Liu, Zheng, Long, Ping, Pan, Tao, Huang, Yuanhua, Li, Yongsheng, Li, Qi, Ma, Yanlin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8900422/
https://www.ncbi.nlm.nih.gov/pubmed/35255977
http://dx.doi.org/10.1186/s13287-022-02768-5