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Gene therapy for severe combined immunodeficiency: are we there yet?

Inherited and acquired diseases of the hematopoietic system can be cured by allogeneic hematopoietic stem cell transplantation. This treatment strategy is highly successful when an HLA-matched sibling donor is available, but if not, few therapeutic options exist. Gene-modified, autologous bone marro...

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Autors principals: Cavazzana-Calvo, Marina, Fischer, Alain
Format: Artigo
Idioma:Inglês
Publicat: American Society for Clinical Investigation 2007
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC1878528/
https://ncbi.nlm.nih.gov/pubmed/17549248
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI30953
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