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Blood Advances|June 6, 2024
A simplified G-CSF-free procedure allows for in vivo HSC gene therapy of sickle cell disease in a mouse modelChang Li, Anna K Anderson, Peter Ruminski, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|March 15, 2026
In vitro and in vivo base editing of CCR5 in hematopoietic stem cells confers HIV-1 resistanceAnna K Anderson, Aphrodite Georgakopoulou, Anne-Sophie Kuhlmann, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|November 3, 2024
Introducing a hemoglobin G-Makassar variant in HSCs by in vivo base editing treats sickle cell disease in miceChang Li, Aphrodite Georgakopoulou, Kiriaki Paschoudi, et al.JCI Insight|August 25, 2022
In vivo base editing by a single i.v. vector injection for treatment of hemoglobinopathiesChang Li, Aphrodite Georgakopoulou, Gregory A Newby, et al.Blood|February 17, 2023
In vivo HSC prime editing rescues sickle cell disease in a mouse modelChang Li, Aphrodite Georgakopoulou, Gregory A Newby, et al.Molecular Therapy. Advances|May 15, 2026
In vivo HSC gene therapy enables sustained eCD4-Ig expression for SIV preventionChang Li, Anna K Anderson, Anne-Sophie Kuhlmann, et al.Pageof 1