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CRISPR/Cas9-generated p47(phox)-deficient cell line for Chronic Granulomatous Disease gene therapy vector development

Development of gene therapy vectors requires cellular models reflecting the genetic background of a disease thus allowing for robust preclinical vector testing. For human p47(phox)-deficient chronic granulomatous disease (CGD) vector testing we generated a cellular model using clustered regularly in...

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Detalles Bibliográficos
Autores principales:	Wrona, Dominik, Siler, Ulrich, Reichenbach, Janine
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group 2017
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5347011/ https://www.ncbi.nlm.nih.gov/pubmed/28287132 http://dx.doi.org/10.1038/srep44187

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