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Gene therapy for PIDs: Progress, pitfalls and prospects
Substantial progress has been made in the past decade in treating several primary immunodeficiency disorders (PIDs) with gene therapy. Current approaches are based on ex-vivo transfer of therapeutic transgene via viral vectors to patient-derived autologous hematopoietic stem cells (HSCs) followed by...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Elsevier/North-Holland
2013
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3725417/ https://www.ncbi.nlm.nih.gov/pubmed/23566838 http://dx.doi.org/10.1016/j.gene.2013.03.098 |