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...
| Príomhchruthaitheoirí: | , , , , , , , , , , |
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| Formáid: | Alt |
| Teanga: | English |
| Foilsithe / Cruthaithe: |
Ferrata Storti Foundation
2014-01-01
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| Sraith: | Haematologica |
| Rochtain ar líne: | https://haematologica.org/article/view/6909 |