New gene therapy model offers hope for X-linked sideroblastic anemia treatment

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Researchers at Children's Hospital of Philadelphia have pioneered a new 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.

New gene therapy model offers hope for X-linked sideroblastic anemia treatment retrieved 14 May 2024 from https://medicalxpress.com/news/2024-05-gene-therapy-linked-sideroblastic-anemia.html

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