Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice

CRISPR/Cas9-mediated beta-globin (HBB) gene correction of sickle cell disease (SCD) patient-derived hematopoietic stem cells (HSCs) in combination with autologous transplantation represents a recent paradigm in gene therapy. Although several Cas9-based HBB-correction approaches have been proposed, f...

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Udgivet i:Nat Commun
Main Authors: Wilkinson, Adam C., Dever, Daniel P., Baik, Ron, Camarena, Joab, Hsu, Ian, Charlesworth, Carsten T., Morita, Chika, Nakauchi, Hiromitsu, Porteus, Matthew H.
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group UK 2021
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7846836/
https://ncbi.nlm.nih.gov/pubmed/33514718
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41467-021-20909-x
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