Gene Technology

Abstract

The Current State-of-the-Art in Therapeutic Genome Editing and the Future

Donya Moradi Manesh and Punam Malik

Genome editing using designer site-specific nucleases is a burgeoning field in which genomes of target cells/ organisms are now being manipulated to create/correct mutations or transcriptionally manipulate gene expression. The field began over a decade ago with zinc finger nucleases, which were soon followed by designer homing endonucleases/mega nucleases, transcription activator-like effector nucleases, and more recently, CRISPR/Cas9. Each platform has its own strengths and weaknesses but they all allow editing of the genome in cells and organisms to either study biology/function of genes or for a therapeutic effect. This review will briefly describe the various gene editing platforms and then focus in on the CRISPR/Cas9 system.