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Nucleic Acid Therapy for β-Thalassemia
β-thalassemia is caused by mutations in the β-globin gene which diminishes or abolishes β-globin chain production. This reduction causes an imbalance of the α/β-globin chain ratio and contributes to the pathogenesis of the disease. Several approaches to reduce the imbalance of the α/β ratio using se...
Autor principal: | d’Arqom, Annette |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Dove
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7501986/ https://www.ncbi.nlm.nih.gov/pubmed/32982166 http://dx.doi.org/10.2147/BTT.S265767 |
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