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The Future of Gene Therapy for Transfusion-Dependent Beta-Thalassemia: The Power of the Lentiviral Vector for Genetically Modified Hematopoietic Stem Cells

β-thalassemia, a disease that results from defects in β-globin synthesis, leads to an imbalance of β- and α-globin chains and an excess of α chains. Defective erythroid maturation, ineffective erythropoiesis, and shortened red blood cell survival are commonly observed in most β-thalassemia patients....

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Detalles Bibliográficos
Autores principales:	Rattananon, Parin, Anurathapan, Usanarat, Bhukhai, Kanit, Hongeng, Suradej
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Frontiers Media S.A. 2021
Materias:	Pharmacology
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8517149/ https://www.ncbi.nlm.nih.gov/pubmed/34658870 http://dx.doi.org/10.3389/fphar.2021.730873

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