Ex-vivo Gene Therapy Restores LEKTI Activity and Corrects the Architecture of Netherton Syndrome-derived Skin Grafts

Netherton syndrome (NS) is a debilitating congenital skin disorder caused by mutations in the SPINK5 gene encoding the lymphoepithelial Kazal-type-related inhibitor (LEKTI). It is characterized by defective keratinization, recurrent infections, and hypernatraemic dehydration with a mortality rate of...

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Detalhes bibliográficos
Main Authors: Di, Wei-Li, Larcher, Fernado, Semenova, Ekaterina, Talbot, Gill E, Harper, John I, Del Rio, Marcela, Thrasher, Adrian J, Qasim, Waseem
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2011
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3034839/
https://ncbi.nlm.nih.gov/pubmed/20877344
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2010.201
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