Article
Targeted Repair of CYBB in X-CGD iPSCs Requires Retention of Intronic Sequences for Expression and Functional Correction.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Feb 2017
Sweeney Colin L, Zou Jizhong, Choi Uimook, Merling Randall K, Liu Alexander, Bodansky Aaron, Burkett Sandra, Kim Jung-Woong, De Ravin Suk See, Malech Harry L
Abstract excerpt
X-linked chronic granulomatous disease (X-CGD) is an immune deficiency resulting from defective production of microbicidal reactive oxygen species (ROS) by phagocytes. Causative mutations occur throughout the CYBB gene, resulting in absent or defective gp91phox protein expression. To correct CYBB exon 5 mutations while retaining normal gene regulation, we utilized TALEN or Cas9 for exon 5 replacement in induced...
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