Genetic Engineering & Biotechnology News

DEC 2017

Genetic Engineering & Biotechnology News (GEN) is the world's most widely read biotech publication. It provides the R&D community with critical information on the tools, technologies, and trends that drive the biotech industry.

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4 | DECEMBER 2017 | GENengnews.com O ne sweltering summer afternoon in 2003, a vacationing Spanish microbiologist named Francisco Mojica told his wife he was going to seek refuge in his air-conditioned lab at the University of Alicante. For years, Mojica had been puzzling over the putative function of a bizarre stretch of DNA that had a funny name (CRISPR) and was found in the genomes of bacteria and other prokaryotes. That afternoon, as he ran yet another computer search, he made a stunning discovery: for the first time, one of these mystery bacterial DNA segments scored a hit—with a virus sequence. The significance of that discovery escaped most scientists and even the editors of several prestigious science journals (including Nature), who passed on the ensuing research paper before it was eventually published in 2005 in a specialist evolution journal. Dr. Mojica and coworkers had stumbled upon an ancient bacterial immune defense system—one that stores bits of viral genetic code—and that functioned as a mugshot, so to speak. Should any virus threaten to become a repeat offender, its mugshot allows it to be more readily identified and apprehended. The role of the CRISPR (Clustered Regularly Inter- spersed Short Palindromic Repeats) motif and its associated nuclease (DNA-cleaving) proteins was soon confirmed experimentally. But as the world now knows, the major breakthrough occurred in 2012, when a collaboration between the labs of Emmanuelle Charpentier, Ph.D., (Max Planck Institute for Infection Biology, Berlin) and Jennifer Doudna, Ph.D. (Howard Hughes Medical Institute, UC Berkeley), adapted the CRISPR system (using a nuclease called Cas9) into a potent gene-editing tool, capable of targeting and cutting virtually any DNA sequence. Once the labs of Feng Zhang, Ph.D., and George Church, Ph.D., demonstrated that CRISPR/Cas9 could edit human genes in vivo, a biotech revolution was unleashed. Five years on, one can hardly pick up a science journal or biotech magazine without reading about another CRISPR-related advance. In the past six months, we have seen major advances in editing disease-causing genes in human embryos. New tools include RNA-editing CRISPR systems and ultra-precise base editors. In the latter, a modified Cas protein pinpoints and tweaks a specific nu- cleotide, rather than completely cleaving the double helix. In this timely supplement, GEN has compiled a selection of topical features on novel applications of CRISPR/Cas9 that neatly capture the incredible excitement and potential of this technology. Kicking things off is Malorye Branca's excellent feature exploring clinical applications of CRISPR therapies entitled: "A Dose of CRISPR: Can Gene-Editing Cut It in the Clinic?" (This originally appeared as a cover story in GEN's sister magazine, Clinical OMICs.) Other articles from GEN in this supplement cover a broad range of issues, including enhancing and scaling up fundamental CRISPR genome-editing technology, and a range of new applications from engineering the pig genome for safer organ transplantation to various novel strategies in gene therapy. We thank all of our sponsors for making this special supplement possible. The explosion of research in CRISPR is as good a reason as any why GEN's publisher, Mary Ann Liebert, Inc., is launching The CRISPR Journal in 2018. (Please visit www.crisprjournal.com for more information.) You can also be sure that GEN and Clinical OMICs will be the first to report on major advances across the CRISPR landscape throughout 2018 and beyond. Peak CRISPR: On the Cusp of a Biomedical Revolution B E S T O F C R I S P R 2 017 John Sterling Editor-in-Chief, GEN Kevin Davies Editor-at-Large, GEN Executive Editor, The CRISPR Journal GENengnews.com

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