Developmental BiologyNature CommunicationsPeer-reviewed
Cas9-AAV6 Gene Correction of Beta-Globin in Autologous HSCs Improves Sickle Cell Disease Erythropoiesis in Mice
Adam C. Wilkinson, Daniel P. Dever, Ron Baik…·Matthew Hebden Porteus
This study corrects the beta-globin gene in sickle cell disease patient-derived HSCs using CRISPR/Cas9 and shows improved in vivo erythropoiesis in a mouse model.
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