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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Dettagli Bibliografici
Pubblicato in:Stem Cell Res Ther
Autori principali: Wattanapanitch, Methichit, Damkham, Nattaya, Potirat, Ponthip, Trakarnsanga, Kongtana, Janan, Montira, U-pratya, Yaowalak, Kheolamai, Pakpoom, Klincumhom, Nuttha, Issaragrisil, Surapol
Natura: Artigo
Lingua:Inglês
Pubblicazione: BioMed Central 2018
Soggetti:
Research
Accesso online: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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