Targeted Repair of CYBB in X-CGD iPSCs Requires Retention of Intronic Sequences for Expression and Functional Correction

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 gp91(phox) protein expression. To correct CYBB...

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Bibliografiske detaljer
Udgivet i:Mol Ther
Main Authors: 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.
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2017
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Original Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5368476/
https://ncbi.nlm.nih.gov/pubmed/28153086
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2016.11.012
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