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First of its kind gene therapy model offers hope for X-linked sideroblastic anemia treatmentand 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 ...
Patients with XLSA experience a disruption in heme synthesis, leading to a spectrum of issues such as severe anemia and iron overload, which can cause symptoms like extreme fatigue, shortness of ...
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