Search for Articles:
Title / Keyword
Author / Affiliation / Email
Journal
Article Type
 
 
Advanced Search
 
Section
Special Issue
Volume
Issue
Number
Page
 
9.9
5.1
Journals
Cells
Special Issues
CRISPR-Based Genome Editing in Translational Research—Third Edition
share announcement

CRISPR-Based Genome Editing in Translational Research—Third Edition

A special issue of Cells (ISSN 2073-4409).

Deadline for manuscript submissions: 30 September 2025 | Viewed by 489

Special Issue Editors

Dr. Jie Xu

E-Mail Website
Guest Editor
Center for Advanced Models and Translational Sciences and Therapeutics, University of Michigan Medical Center, Ann Arbor, MI 48109, USA
Interests: genome; gene editing; cell biology; CRISPR/Cas9
Special Issues, Collections and Topics in MDPI journals
Dr. Dongshan Yang

E-Mail Website
Guest Editor
Center for Advanced Models and Translational Sciences and Therapeutics, University of Michigan Medical Center, Ann Arbor, MI 48109, USA
Interests: genome; gene editing; cell biology; CRISPR/Cas9
Special Issues, Collections and Topics in MDPI journals
Dr. Jifeng Zhang

E-Mail Website
Guest Editor
Center for Advanced Models for Translational Sciences and Therapeutics, University of Michigan Medical School, Ann Arbor, MI 48109, USA
Interests: genome; gene editing; cell biology; CRISPR/Cas9
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

This is the expanded second edition of ‘CRISPR-Based Genome Editing in Translational Research—Second Edition’, the edition of which led to the publication of seven papers.

Emerging gene editing tools represented by CRISPR/Cas9 have had a significant impact on the translational biomedical research field. Thanks to their ease of use and high efficiency, they are widely used for the production of novel animal and cellular models and for the development of gene-editing-based therapy for genetic and non-genetic diseases. At the same time, many roadblocks remain in the path toward their eventual clinical applications, such as substantial off-target editing events which need to be minimized and a lack of efficient and safe in vivo delivery methods.

This Special Issue calls for research and review articles on any topics related to the translational application of CRISPR in modern biomedical research.

Dr. Jie Xu
Dr. Dongshan Yang
Dr. Jifeng Zhang
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. 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.

Keywords

  • genome
  • gene editing
  • CRISPR/Cas9

Benefits of Publishing in a Special Issue

  • Ease of navigation: Grouping papers by topic helps scholars navigate broad scope journals more efficiently.
  • Greater discoverability: Special Issues support the reach and impact of scientific research. Articles in Special Issues are more discoverable and cited more frequently.
  • Expansion of research network: Special Issues facilitate connections among authors, fostering scientific collaborations.
  • External promotion: Articles in Special Issues are often promoted through the journal's social media, increasing their visibility.
  • e-Book format: Special Issues with more than 10 articles can be published as dedicated e-books, ensuring wide and rapid dissemination.

Further information on MDPI's Special Issue policies can be found here.

Related Special Issue

Published Papers (1 paper)

Order results
Result details
Show export options expand_more Show export options expand_less
Select all
Export citation of selected articles as:

Research

22 pages, 8953 KiB  
Article
Investigation on ABCC6-Deficient Human Hepatocytes Generated by CRISPR–Cas9 Genome Editing
by Ricarda Plümers, Svenja Jelinek, Christopher Lindenkamp, Michel R. Osterhage, Cornelius Knabbe and Doris Hendig
Cells 2025, 14(8), 576; https://doi.org/10.3390/cells14080576 - 11 Apr 2025
Viewed by 249
Abstract
Patients affected by the rare disease pseudoxanthoma elasticum (PXE) exhibit the calcification of elastic fibers in ocular, dermal, and vascular tissues. These symptoms are triggered by mutations in the ATP-binding cassette transporter subfamily C member 6 (ABCC6), whose substrate remains unknown. Interestingly, ABCC6 [...] Read more.
Patients affected by the rare disease pseudoxanthoma elasticum (PXE) exhibit the calcification of elastic fibers in ocular, dermal, and vascular tissues. These symptoms are triggered by mutations in the ATP-binding cassette transporter subfamily C member 6 (ABCC6), whose substrate remains unknown. Interestingly, ABCC6 is predominantly expressed in the liver tissue, leading to the hypothesis that PXE is a metabolic disorder. We developed a genome-editing system targeting ABCC6 in human immortalized hepatocytes (HepIms) for further investigations. The HepIms were transfected with an ABCC6-specific clustered regulatory interspaced short palindromic repeat (CRISPR-Cas9) genome-editing plasmid, resulting in the identification of a heterozygous (htABCC6HepIm) and a compound heterozygous (chtABCC6HepIm) clone. These clones were analyzed for key markers associated with the PXE pathobiochemistry. Hints of impaired lipid trafficking, defects in the extracellular matrix remodeling, the induction of calcification inhibitor expression, and the down regulation of senescence and inflammatory markers in ABCC6-deficienct HepIms were found. Our ABCC6 knock-out model of HepIms provides a valuable tool for studying the metabolic characteristics of PXE in vitro. The initial analysis of the clones mirrors various features of the PXE pathobiochemistry and provides an outlook on future research approaches. Full article
(This article belongs to the Special Issue CRISPR-Based Genome Editing in Translational Research—Third Edition)
►▼ Show Figures

Figure 1

Show export options expand_more Show export options expand_less
Select all
Export citation of selected articles as:
 
Displaying articles 1-1
Back to TopTop