VIDEO: Improving function of factor VIII variants for hemophilia A gene therapy
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In this video, Lindsey A. George, MD, talked about improving the function of factor VIII variants for hemophilia A gene therapy, which she presented in a session at ASH Annual Meeting and Exposition.
George, assistant professor of pediatrics at the Perelman School of Medicine at the University of Pennsylvania, and director of clinical in vivo gene therapy and attending hematologist at the Children’s Hospital of Philadelphia, presented all the proof-of-concept efficacy and safety studies that will hopefully support therapeutic translation in terms of developing “initial efforts” in hemophilia A and B gene editing.
“We’re excited about that, but we’ll ultimately see how this may fare potentially in clinical translation,” George said.
Reference:
George LA, et al. Rational design of factor VIII variants for hemophilia A gene therapy. Presented at: ASH Annual Meeting and Exposition; Dec. 9-12, 2023; San Diego.
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