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Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia
β-Thalassemia is one of the most common inherited anemias, with no effective cure for most patients. The pathophysiology reflects an imbalance between α- and β-globin chains with an excess of free α-globin chains causing ineffective erythropoiesis and hemolysis. When α-thalassemia is co-inherited wi...
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| Udgivet i: | Nat Commun |
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| Main Authors: | , , , , , , , , , , , , , , , , , , , , , |
| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
Nature Publishing Group UK
2017
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5583283/ https://ncbi.nlm.nih.gov/pubmed/28871148 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41467-017-00479-7 |
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