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Erythroid-specific Expression of β-globin by Sleeping Beauty Transposon for Sickle Cell Disease
Sickle cell disease (SCD) results predominately from a single monogenic mutation that affects thousands of individuals worldwide. Gene therapy approaches have focused on using viral vectors to transfer wild type β- or γ-globin transgenes into hematopoietic stem cells for long-term expression of the...
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| Главные авторы: | , , , , , |
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| Формат: | Artigo |
| Язык: | Inglês |
| Опубликовано: |
2007
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| Предметы: | |
| Online-ссылка: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3893920/ https://ncbi.nlm.nih.gov/pubmed/17508724 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1021/bi6024484 |
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