One-step genetic correction of hemoglobin E/beta-thalassemia patient-derived iPSCs by the CRISPR/Cas9 system
Abstract 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-matche...
Main Authors: | Methichit Wattanapanitch, Nattaya Damkham, Ponthip Potirat, Kongtana Trakarnsanga, Montira Janan, Yaowalak U-pratya, Pakpoom Kheolamai, Nuttha Klincumhom, Surapol Issaragrisil |
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Format: | Article |
Language: | English |
Published: |
BMC
2018-02-01
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Series: | Stem Cell Research & Therapy |
Subjects: | |
Online Access: | http://link.springer.com/article/10.1186/s13287-018-0779-3 |
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