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A preclinical approach for gene therapy of β-thalassemia
Lentiviral-mediated β-globin gene transfer successfully treated β-thalassemic mice. Based on this result, clinical trials were initiated. To date, however, no study has investigated the efficacy of gene therapy in relation to the nature of the different β-globin mutations found in patients. Most mut...
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| Hlavní autoři: | , , , , , , , , , |
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| Médium: | Artigo |
| Jazyk: | Inglês |
| Vydáno: |
2010
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| Témata: | |
| On-line přístup: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3068625/ https://ncbi.nlm.nih.gov/pubmed/20712784 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1111/j.1749-6632.2010.05594.x |
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