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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Dades bibliogràfiques
Publicat a:Stem Cell Res Ther
Autors principals: Wattanapanitch, Methichit, Damkham, Nattaya, Potirat, Ponthip, Trakarnsanga, Kongtana, Janan, Montira, U-pratya, Yaowalak, Kheolamai, Pakpoom, Klincumhom, Nuttha, Issaragrisil, Surapol
Format: Artigo
Idioma:Inglês
Publicat: BioMed Central 2018
Matèries:
Research
Accés en línia: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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