News Release 

Media alert: new articles in The CRISPR Journal

Mary Ann Liebert, Inc./Genetic Engineering News

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IMAGE: The CRISPR Journal is dedicated to validating and publishing outstanding research and commentary on all aspects of CRISPR and gene editing, including CRISPR biology, technology, and genome editing, and commentary... view more 

Credit: Mary Ann Liebert, Inc., publishers

The CRISPR Journal announces the publication of its April 2019 issue. The Journal is dedicated to validating and publishing outstanding research and commentary on all aspects of CRISPR and gene editing, including CRISPR biology, technology, and genome editing, and commentary and debate of key policy, regulatory, and ethical issues affecting the field. The Journal, led by Editor-in-Chief Rodolphe Barrangou, PhD (North Carolina State University) and Executive Editor Dr. Kevin Davies, is published bimonthly in print and online. See http://www.crisprjournal.com for more information.

This press release is copyright Mary Ann Liebert, Inc. Its use is granted only for journalists and news media receiving it directly from The CRISPR Journal. For full-text copies of articles or to arrange interviews with Dr. Barrangou, Dr. Davies, authors, or members of the editorial board, contact Kathryn Ryan at the Publisher.

1. CRISPR-Cas9-mediated editing to correct a kidney disease causing mutation

A genetic disorder called focal segmental glomerulosclerosis (FSGS) can cause nephrotic syndrome in children and is a leading cause of kidney failure in adults. A missense mutation in the PAX2 gene, which encodes a transcription factor, was recently identified in a family with adult-onset autosomal dominant FSGS. In a new study, scientists from the Mario Negri Institute for Pharmacological Research successfully corrected the missense mutation in PAX2 in patient-derived induced pluripotent stem cells (iPSCs). This establishes a renewable source of the PAX2-/- iPSC cells with the patient-specific genetic background. This disease model makes it possible to dissect the altered molecular mechanisms causing FSGS and provides significant technical advances in studies into the role of other FSGS-associated genetic abnormalities.

Contact: Susanna Tomasoni (Mario Negri Institute for Pharmacological Research, Italy)

2. The Birth of Base Editing: Alexis Komor and Nicole Gaudelli (Interview)

In 2013, graduate student Alexis Komor exchanged a series of emails with Harvard University chemist David Liu, her future postdoc supervisor, outlining potential schemes to engineer base changes in DNA. Komor led the development of the first base editor - a molecular machine that catalyzes the transition of a cytosine to a thymine nucleotide. Her friend and colleague, Nicole Gaudelli, followed that work by developing a companion base editor that can engineer adenine to guanine substitutions. In this interview, Komor (now an assistant professor at University of California, San Diego) and Gaudelli (senior scientist, Beam Therapeutics) exclusively share stories of the ingenuity, tenacity, and serendipity that spurred the development of these powerful gene editing systems.

Contact: Alexis Komor (UCSD) and Nicole Gaudelli (Beam Therapeutics)

Note: The audio of this interview was released in an episode of GuidePost on April 10, 2019.

3. Base Editors: CRISPR-based editing without the break (Review)

The CRISPR-Cas system has revolutionized genome editing over the past 5-6 years, but a new method of genome editing - base editing - offers great promise in engineering precise base substitutions, without having to cleave the DNA. By combining enzymes that modify DNA bases with catalytically inactive Cas variants, nucleotide conversion can be achieved without the need for double-stranded breaks or a donor template. In a review article, Chinese scientists led by Jia Chen at ShanghaiTech University discuss base editing fundamentals, the development and the application of base editors, and the potential future usage of this transformative technology.

Contact: Jia Chen (ShanghaiTech University, China)

4. A novel CRISPR-based method for multiplex site-directed mutagenesis

Much of our understanding of gene function stems from the study of their mutated counterparts. Often, a PCR-based site-directed mutagenesis (SDM) technique is employed to engineer these variants. While these tools are efficient, they carry limitations, such as off-site mutagenesis, limited scalability and lack of multiplexing capabilities. In a follow-up study to work published in The CRISPR Journal last April, Eric Kmiec and colleagues use the same mammalian cell-free system to catalyze DNA fragment replacement using a CRISPR-Cas12a dual cleavage mechanism to examine the conditions that lead to gene/fragment replacement. Termed CRISPR-directed DNA mutagenesis (CDM), the authors produce molecules with multiple site-specific mutations and site-specific deletions in the same reaction mixture. The method, by comparison to SDM, produces fewer unintended mutations in the target gene.

Contact: Eric Kmiec (University of Delaware; Gene Editing Institute, Christiana Care Health System)

Also in this issue:

5. CRISPR at the Cinema

An early review of Human Nature, the CRISPR documentary directed by Adam Bolt and produced by Elliot Kirschner and Dan Rather, currently playing on the festival circuit.

Contact: Courtney Klotz (Managing Editor, The CRISPR Journal)

6. Chipping in on diagnostics (First Cut)

Two members of the Feng Zhang laboratory at MIT, Omar Abudayyeh and Jonathan Gootenberg, discuss the recent report of a CRISPR-chip, a graphene-based field-effect transistor device for rapid and precise diagnostic applications.

Contact: Omar Abudayyeh and Jonathan Gootenberg (McGovern Institute for Brain Research, MIT)

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