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Gaining the hard yard: pre-clinical evaluation of lentiviral-mediated gene therapy for the treatment of β-thalassemia
Gene therapy is one potential novel therapeutic avenue for the treatment of inherited monogenic disorders. Diseases of the blood are frequent targets for gene therapy because it is relatively easy to harvest haematopoietic stem cells (HSCs) from the bone marrow, genetically modify the cells ex vivo,...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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WILEY-VCH Verlag
2010
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3377332/ https://www.ncbi.nlm.nih.gov/pubmed/20677209 http://dx.doi.org/10.1002/emmm.201000086 |