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Targeted genome editing tools for disease modeling and gene therapy

Cai, Mi and Yang, Yi (2013) Targeted genome editing tools for disease modeling and gene therapy. Current Gene Therapy.


The rapid advancement of next generation sequencing (NGS) technology has made it possible to interrogate patients’ genome and transcriptome at unprecedented depth. How to quickly and efficiently translate the wealth of genomic information into clinical benefits has remained challenging for pharmaceutical industry. Targeted genome editing plays a crucial role in dissecting out disease-related genotypes. Once only available for a few cell types and model organisms, its application has been greatly broadened since the introduction of custom designed nucleases (CDNs), including zinc finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs) which generate a targeted DNA double-strand break (DSB) in the genome. Once a DSB is created, specific modifications can be introduced around the breakage site during its repair. Recently, clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) system has been shown to be more efficient and flexible than CDNs for gene targeting in multiple cell lines and organisms. Here, we review the progress that has been made for all three genome editing technologies in modifying both cells and model organisms, compare important aspects of each approach, and summarize the applications of these tools in disease modeling and gene therapy, and in the end, we discuss future prospects of the field.

Item Type: Article
Date Deposited: 13 Oct 2015 13:13
Last Modified: 13 Oct 2015 13:13