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

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...

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Main Authors: Methichit Wattanapanitch, Nattaya Damkham, Ponthip Potirat, Kongtana Trakarnsanga, Montira Janan, Yaowalak U-pratya, Pakpoom Kheolamai, Nuttha Klincumhom, Surapol Issaragrisil
Format: Article
Language:English
Published: BMC 2018-02-01
Series:Stem Cell Research & Therapy
Subjects:
Induced pluripotent stem cells
Thalassemia
Hematopoietic differentiation
Genetic correction
CRISPR/Cas9
Online Access:http://link.springer.com/article/10.1186/s13287-018-0779-3

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http://link.springer.com/article/10.1186/s13287-018-0779-3

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