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A Novel Model of X-Linked Sideroblastic Anemia (Alas2-Ko, XLSA) with Ring Sideroblasts, Severe Anemia, and Iron Overload is Rescued by Lentiviral Gene Transfer

Carlo Castruccio Castracani,Amaliris Guerra,Laura Breda,Tyler Papp,Enrico Radaelli,Charles-Antoine Assenmacher, Giovanni Finesso, Yink Tam,Barbara Mui,Simona Fontana,Chiara Riganti,Veronica Fiorito,Sara Petrillo,Emanuela Tolosano,Hamideh Parhiz,Stefano Rivella

MOLECULAR THERAPY（2024）

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