Chemical Control of Genome Editing

Dear Colleagues,

The breakthrough technology of gene editing, and in particular, CRISPR/Cas has revolutionized the potential for genomic modification in human cells. This exciting technology already plays an important role in drug discovery, agriculture and the nascent field of gene drives. The potential for clinical application is developing at a rapid rate, and a number of clinical trials are already in the making.  The possibility for curing diseases appears to be just around the corner. Yet, unregulated activity of CRISPR/Cas, as well as the handful of other gene editing techniques can lead to unwanted and potentially serious outcomes. In this important and timely issue of Molecules, we will examine the use of small molecules to control the regulatory circuitry that surrounds gene editing in human cells. Topics of interest include small molecules that enhance nuclear delivery, those that tip the balance toward homology directed repair and away from nonhomologous end joining, potential approaches that act to modify the Cas nuclease family both directly and indirectly and molecules that act as anti-CRISPR agents for the overall control of chromosomal modification. Small molecules or strategies that reduce off-site mutagenesis or regulate the amount of indel formation surrounding the target site are also welcome and of great interest.

Dr. Clifford J. Steer
Dr. Eric B. Kmiec
Guest Editors

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Molecules is an international peer-reviewed open access semimonthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2700 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

• genome editing
• small molecules
• CRISPR/Cas
• nuclear delivery
• functional regulation