Gene-edited pseudogene resurrection corrects p47phox-deficient chronic granulomatous disease

作者：Randall K. Merling, Douglas B. Kuhns, Colin L. Sweeney, Xiaolin Wu, Sandra Burkett, Jessica Chu, Janet Lee, Sherry Koontz, Giovanni Di Pasquale, Sandra Afione, John A. Chiorini, Elizabeth M. Kang, Uimook Choi, Suk See De Ravin, Harry L. Malech · 发表于：Blood Advances · 年份：2016 · DOI：10.1182/bloodadvances.2016001214 · 被引用次数：49 · 研究领域：CRISPR and Genetic Engineering、Neutrophil, Myeloperoxidase and Oxidative Mechanisms、Advanced biosensing and bioanalysis techniques

Key Points Gene-editing correction of the GT deletion in exon 2 of NCF1 pseudogenes corrects p47phox-deficient chronic granulomatous disease. The nonfunctional pseudogenes NCF1B and NCF1C can be resurrected to produce functional p47phox protein by gene editing.