Multiplex CRISPR/Cas9 genome editing in hematopoietic stem cells for fetal hemoglobin reinduction generates chromosomal translocations
Sickle cell disease and β-thalassemia are common monogenic disorders that cause significant morbidity and mortality globally. The only curative treatment currently is allogeneic hematopoietic stem cell transplantation, which is unavailable to many patients due to a lack of matched donors and carries...
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| Auteurs principaux: | , , , , , , , , , |
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| Format: | article |
| Langue: | EN |
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Elsevier
2021
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| Accès en ligne: | https://doaj.org/article/c920282311d94065aac1d4319ebc7ffb |
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