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

© 2018 The Author(s). 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...

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Bibliographic Details
Main Authors: Methichit Wattanapanitch, Nattaya Damkham, Ponthip Potirat, Kongtana Trakarnsanga, Montira Janan, Yaowalak U-Pratya, Pakpoom Kheolamai, Nuttha Klincumhom, Surapol Issaragrisil
Other Authors: Chulalongkorn University
Format: Article
Published: 2019
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Online Access:https://repository.li.mahidol.ac.th/handle/123456789/45238
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Institution: Mahidol University