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[해외논문] Gene Therapy for Hemoglobinopathies

Human gene therapy, v.34 no.17/18, 2023년, pp.793 - 807  

Lidonnici, Maria Rosa (San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), San Raffaele Scientific Institute, Milan, Italy) ,  Scaramuzza, Samantha (and) ,  Ferrari, Giuliana (San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), San Raffaele Scientific Institute, Milan, Italy)

Abstract AI-Helper 아이콘AI-Helper

β-Thalassemia and sickle cell disease are autosomal recessive disorders of red blood cells due to mutations in the adult β-globin gene, with a worldwide diffusion. The severe forms of hemoglobinopathies are fatal if untreated, and allogeneic bone marrow transplantation can be offered to a ...

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참고문헌 (137)

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