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

by Adam C. Wilkinson, Daniel P. Dever, Ron Baik, Joab Camarena, Ian Hsu, Carsten T. Charlesworth, Chika Morita, Hiromitsu Nakauchi, Matthew H. Porteus
Published 2021-01-01

Priming Human Repopulating Hematopoietic Stem and Progenitor Cells for Cas9/sgRNA Gene Targeting

by Carsten T. Charlesworth, Joab Camarena, M. Kyle Cromer, Sriram Vaidyanathan, Rasmus O. Bak, Jason M. Carte, Jason Potter, Daniel P. Dever, Matthew H. Porteus
Published 2018-09-01