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Development of a Lentiviral Gene Therapy for Alpha Thalassemia

Donald B. Kohn &
Eva Segura, PhD Candidate

Tuesday, November 7, 2023, 8:00-9:00 am (San Francisco)

ATM Quarterly Meeting-Nov 7, 2023, Lentiviral Gene Therapy for Alpha Thalassemia Event-ATM Qrtly-Nov 7, 2023

In this session:

  • An overview of gene therapy
  • Beta globin lentiviral vectors developed for sickle cell disease
  • Development of alpha globin lentiviral vectors for alpha thalassemia major (ATM) based on a clinical beta globin vectors
  • Assessment of candidate lentiviral vectors in healthy donor and ATM patient hematopoietic stem and progenitor cells derived into red blood cells