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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Detalhes bibliográficos
Publicado no:Stem Cell Res Ther
Main Authors: Wattanapanitch, Methichit, Damkham, Nattaya, Potirat, Ponthip, Trakarnsanga, Kongtana, Janan, Montira, U-pratya, Yaowalak, Kheolamai, Pakpoom, Klincumhom, Nuttha, Issaragrisil, Surapol
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2018
Assuntos:
Research
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5828150/
https://ncbi.nlm.nih.gov/pubmed/29482624
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13287-018-0779-3
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