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First of Its Kind Gene Therapy Model Offers Hope for X-Linked Sideroblastic Anemia Treatment

Researchers at Children's Hospital of Philadelphia (CHOP) and the University of Pennsylvania Perelman School of Medicine pioneered a first of its kind gene therapy model that offers a potential breakthrough in treating X-linked sideroblastic anemia (XLSA), a rare congenital anemia caused by mutations in the ALAS2 gene crucial for the synthesis of heme, a key compound in hemoglobin.

January 2, 2025


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Angell Street Psychiatry
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