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CRISPR-based genome editing technologies poised to revolutionize medicine and industry

Published: Monday, July 20, 2015 - 13:03 in Biology & Nature

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<p><a target="_blank" href="http://www.liebertpub.com/hgt"><em>Human Gene Therapy</em></a>, the Official Journal of the European Society of Gene and Cell Therapy, British Society for Gene and Cell Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies, is an authoritative peer-reviewed journal published monthly in print and online. Led by Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, <em>Human Gene Therapy</em> presents reports on the transfer and expression of genes in mammals, including humans. Related topics include improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, as well as ethical, legal, and regulatory issues related to the gene transfer in humans. Its companion journals, <em>Human Gene Therapy Methods</em>, published bimonthly, focuses on the application of gene therapy to product testing and development, and <em>Human Gene Therapy Clinical Development</em>, published quarterly, features data relevant to the regulatory review and commercial development of cell and gene therapy products. Tables of contents for all three publications and a free sample issue may be viewed on the <em>Human Gene Therapy</em> website.

©Mary Ann Liebert, Inc., publishers

CRISPR/Cas systems for genome editing have revolutionized biological research over the past three years, and their ability to make targeted changes in DNA sequences in living cells with relative ease and affordability is now being applied to clinical medicine and will have a significant impact on advances in drug and other therapies, agriculture, and food products. The power and promise of this innovation are presented in the Review article "The Bacterial Origins of the CRISPR Genome-Editing Revolution," published in a special issue of Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Human Gene Therapy website until October 15.2015. Erik Sontheimer, University of Massachusetts Medical School, Worcester, and Rodolphe Barrangou, North Carolina State University, Raleigh, describe the origins of this technology, which were derived from DNA sequences found in many bacteria known as clustered, regularly interspaced, short palindromic repeats (CRISPR) regions. These are part of bacteria's protective immune system. They have been developed into genome editing tools comprised of a "hardware" component (an RNA-guided DNA-targeting system that breaks a DNA strand at a specific site, with the help of the Cas protein), and a "software" component that can be programmed, and re-programmed, to repair or replace a faulty gene.