Genetic Modulation of RNA Splicing with a CRISPR-Guided Cytidine Deaminase

Summary: This protocol uses lipofectamine to deliver base editors (i.e., dCas9 and AIDx fusion protein) and sgRNA expression vectors into Duchenne Muscular Dystrophy (DMD) patient-derived human induced pluripotent stem cells (hiPSCs). This protocol details mutation of the 5′ splice site of DMD exon5...

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Bibliographic Details
Main Authors: Yunqing Ma, Juanjuan Yuan, Xing Chang
Format: Article
Language:English
Published: Elsevier 2020-06-01
Series:STAR Protocols
Online Access:http://www.sciencedirect.com/science/article/pii/S266616671930005X