Preclinical Efficacy of a Modified Gamma Globin Lentiviral Vector Mediated Gene Therapy in Berkeley Sickle Cell Anemia Mouse and Human Xenograft Models

Preclinical batches of GbGM and GbGMI lentiviral vector showed both were highly efficacious in correcting sickle cell anemia (SCA) in mice, with sustained gene transfer in primary transplanted SCA mice and high HSC transduction in colony forming units-spleen in secondary transplanted mice.