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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Détails bibliographiques
Auteurs principaux: Clare Samuelson, Stefan Radtke, Haiying Zhu, Mallory Llewellyn, Emily Fields, Savannah Cook, Meei-Li W. Huang, Keith R. Jerome, Hans-Peter Kiem, Olivier Humbert
Format: article
Langue:EN
Publié: Elsevier 2021
Sujets:
HBG
Accès en ligne:https://doaj.org/article/c920282311d94065aac1d4319ebc7ffb
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