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In vivo activation of the human δ-globin gene: the therapeutic potential in β-thalassemic mice
β-thalassemia and sickle cell disease are widespread fatal genetic diseases. None of the existing clinical treatments provides a solution for all patients. Two main strategies for treatment are currently being investigated: (i) gene transfer of a normal β-globin gene; (ii) reactivation of the endoge...
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| Main Authors: | , , , , , , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Ferrata Storti Foundation
2014
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4007936/ https://ncbi.nlm.nih.gov/pubmed/23872310 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3324/haematol.2012.082768 |
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