1. 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 (February 2017)
DOI: /j.ymthe
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2. 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  , DOI: ( /j.ymthe )
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