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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| Main Authors: | , , , , , , , , |
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| Other Authors: | |
| Format: | Article |
| Published: |
2019
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| Subjects: | |
| Online Access: | https://repository.li.mahidol.ac.th/handle/123456789/45238 |
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| Institution: | Mahidol University |