Giorgia Santilli, Adrian J. Thrasher Molecular Therapy

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Presentation transcript:

A New Chapter on Targeted Gene Insertion for X-CGD: Do Not Skip the Intro(n) Giorgia Santilli, Adrian J. Thrasher Molecular Therapy Volume 25, Issue 2, Pages 307-309 (February 2017) DOI: 10.1016/j.ymthe.2017.01.002 Copyright © 2017 Terms and Conditions

Figure 1 Overview of the Gene-Targeting Strategies Devised to Complement CYBB Gene Mutations in iPSCs from Patients with X-CGD (A) TALEN-mediated insertion of a complete CYBB minigene (exon 1–13) in exon 1 fails to restore gp91phox expression in X-CGD iPSC-derived granulocytes. (B) Rescue of the gp91phox protein expression is achieved by CRISPR/Cas9-mediated insertion of the CYBB (exon 2–13) minigene in exon 2. Molecular Therapy 2017 25, 307-309DOI: (10.1016/j.ymthe.2017.01.002) Copyright © 2017 Terms and Conditions