One-step genetic correction of hemoglobin E/beta-thalassemia patient-derived iPSCs by the CRISPR/Cas9 system

BACKGROUND: Thalassemia is the most common genetic disease worldwide; those with severe disease require lifelong blood transfusion and iron chelation therapy. The definitive cure for thalassemia is allogeneic hematopoietic stem cell transplantation, which is limited due to lack of HLA-matched donors...

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Detalles Bibliográficos
Autores principales: Wattanapanitch, Methichit, Damkham, Nattaya, Potirat, Ponthip, Trakarnsanga, Kongtana, Janan, Montira, U-pratya, Yaowalak, Kheolamai, Pakpoom, Klincumhom, Nuttha, Issaragrisil, Surapol
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2018
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5828150/
https://www.ncbi.nlm.nih.gov/pubmed/29482624
http://dx.doi.org/10.1186/s13287-018-0779-3

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