alexa The Current State-of-the-Art in Therapeutic Genome Editing and the Future
ISSN: 2329-6682

Gene Technology
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Research Article

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

Donya Moradi Manesh* and Punam Malik
Division of Experimental Hematology and Cancer Biology and Hematology, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, 3333 Burnet Ave, Cincinnati, OH 45229-3039, USA
*Corresponding Author : Donya Moradi Manesh
Division of Experimental Hematology and Cancer Biology and Hematology
Cancer and Blood Diseases Institute
Cincinnati Children's Hospital Medical Center
Burnet Ave, Cincinnati, USA
Tel: +1 513-636-4200
E-mail: [email protected]
Received: December 07, 2015 Accepted: February 18, 2016 Published: February 21, 2016
Citation: Manesh DM, Malik P (2016) The Current State-of-the-Art in Therapeutic Genome Editing and the Future. Gene Technol 5:135. doi:10.4172/2329-6682.1000135
Copyright: © 2016 Manesh DM, et al. This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.

Abstract

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.

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