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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...

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Detalles Bibliográficos
Autores principales: Mukherjee, Sayandip, Thrasher, Adrian J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier/North-Holland 2013
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