Programmable Molecular Scissors: Applications of a New Tool for Genome Editing in Biotech

Mol Ther Nucleic Acids. 2019 Mar 1:14:212-238. doi: 10.1016/j.omtn.2018.11.016. Epub 2018 Dec 6.

Abstract

Targeted genome editing is an advanced technique that enables precise modification of the nucleic acid sequences in a genome. Genome editing is typically performed using tools, such as molecular scissors, to cut a defined location in a specific gene. Genome editing has impacted various fields of biotechnology, such as agriculture; biopharmaceutical production; studies on the structure, regulation, and function of the genome; and the creation of transgenic organisms and cell lines. Although genome editing is used frequently, it has several limitations. Here, we provide an overview of well-studied genome-editing nucleases, including single-stranded oligodeoxynucleotides (ssODNs), transcription activator-like effector nucleases (TALENs), zinc-finger nucleases (ZFNs), and CRISPR-Cas9 RNA-guided nucleases (CRISPR-Cas9). To this end, we describe the progress toward editable nuclease-based therapies and discuss the minimization of off-target mutagenesis. Future prospects of this challenging scientific field are also discussed.

Keywords: CRISPR-Cas9; DSB; HDR; NHEJ; TALENs; ZFNs; genome editing; nucleases; off-target mutagenesis; ssODNs.

Publication types

  • Review