Targeted Repair of p47-CGD in iPSCs by CRISPR/Cas9: Functional Correction without Cleavage in the Highly Homologous Pseudogenes

Mutations in the NADPH oxidase, which is crucial for the respiratory burst in phagocytes, result in chronic granulomatous disease (CGD). The only curative treatment option for CGD patients, who suffer from severe infections, is allogeneic bone marrow transplantation. Over 90% of patients with mutati...

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Detalles Bibliográficos
Autores principales: Klatt, Denise, Cheng, Erica, Philipp, Friederike, Selich, Anton, Dahlke, Julia, Schmidt, Reinhold E., Schott, Juliane W., Büning, Hildegard, Hoffmann, Dirk, Thrasher, Adrian J., Schambach, Axel
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2019
Materias:
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Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6829751/
https://www.ncbi.nlm.nih.gov/pubmed/31543470
http://dx.doi.org/10.1016/j.stemcr.2019.08.008

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