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Expert Interview

Slingshot members are talking to an expert! The topic is:

A Deep-Dive into Zynteglo as a treatment for beta thalassemia

Ticker(s): BLUE

Who's the expert?

Institution: CHOP

  • Professor of Pediatrics at the Children's Hospital of Philadelphia (CHoP), Chair on Sickle Cell Anemia, & Scientific director of the Comprehensive Center for the Cure of Sickle Cell Disease and Other Red Blood Cell Disorders (CuRED) at CHOP.
  • Research focuses on the pathophysiology and genetics of several murine models of hematopoiesis-, inflammation-, and iron-related disorders as well as in the use of lentiviral vectors for modulation of gene expression and gene transfer for the cure of hemoglobinopathies. 
  • Characterized the role of seminal factors contributing to the morbidity and mortality in beta-thalassemia, sickle cell anemia, Polycythemia vera, hemochromatosis and in anemia of inflammation, such as such hepcidin, intereleukin-6 and ferroportin, the phosphokinase Jak2 and macrophages.

Interview Goal
Discussing the potential of Zynteglo (betibeglogene autotemcel), the recently approved cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia.

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