X - CGD iPSCs 中 CYBB 的靶向修复需要保留内含子序列以进行表达和功能校正。
关键词: CRISPR TALEN X-linked chronic granulomatous disease iPSC intron targeted correction
Mesh : Cell Differentiation / genetics Cell Line Exons Gene Editing Gene Expression Regulation Gene Order Gene Targeting Gene Transfer Techniques Genetic Loci Genetic Vectors Granulocytes / cytology metabolism Granulomatous Disease, Chronic / genetics metabolism therapy Humans Induced Pluripotent Stem Cells / metabolism Introns Membrane Glycoproteins / genetics Mutation NADPH Oxidase 2 NADPH Oxidases / genetics Targeted Gene Repair Transgenes
来 源:
DOI:10.1016/j.ymthe.2016.11.012
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