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Dear Colleagues,

With the rapid evolution of site-specific nuclease systems, such as CRISPR-Cas9 and TALEN, genome editing technology has led to a big breakthrough in the field of life science. Precision gene engineering has been achieved in various cells, animals, plants, and microorganisms, and the derivative technologies have been developed and applied in various ways, including transcriptional control, epigenome editing, chromosome visualization, genome-wide screening, and DNA barcoding.

This Special Issue covers original research and review papers involved in this type of genome editing and related technologies. A wide range of research topics is acceptable, such as the basic development of genome editing tools and methods, functional genomics studies, and biomedical applications using CRISPR-Cas9.

Dr. Tetsushi Sakuma
Guest Editor

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. Cells 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.

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Published Papers (1 paper)

Review

0 pages, 1086 KiB

Open AccessReview

Prime Editing for Human Gene Therapy: Where Are We Now?

by Kelly Godbout and Jacques P. Tremblay

Cells 2023, 12(4), 536; https://doi.org/10.3390/cells12040536 - 7 Feb 2023

Cited by 13 | Viewed by 7876

Abstract

Gene therapy holds tremendous potential in the treatment of inherited diseases. Unlike traditional medicines, which only treat the symptoms, gene therapy has the potential to cure the disease by addressing the root of the problem: genetic mutations. The discovery of CRISPR/Cas9 in 2012 paved the way for the development of those therapies. Improvement of this system led to the recent development of an outstanding technology called prime editing. This system can introduce targeted insertions, deletions, and all 12 possible base-to-base conversions in the human genome. Since the first publication on prime editing in 2019, groups all around the world have worked on this promising technology to develop a treatment for genetic diseases. To date, prime editing has been attempted in preclinical studies for liver, eye, skin, muscular, and neurodegenerative hereditary diseases, in addition to cystic fibrosis, beta-thalassemia, X-linked severe combined immunodeficiency, and cancer. In this review, we portrayed where we are now on prime editing for human gene therapy and outlined the best strategies for correcting pathogenic mutations by prime editing. Full article

(This article belongs to the Special Issue Genome Editing Systems, Methods, Techniques and Their Application Series 3)

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