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

Summary by Stemwell Science News
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. [Molecular Therapy-Methods & Clinical Development] AbstractFull ArticleGraphical Abstract
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Stemwell Science News broke the news in on Friday, February 21, 2025.
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