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Cells
Special Issues
CRISPR-Based Genome Editing in Translational Research—Third Edition
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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 979

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

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Related Special Issue

Published Papers (2 papers)

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Research

14 pages, 3280 KiB  
Article
Validation of Clinical-Grade Electroporation Systems for CRISPR-Cas9-Mediated Gene Therapy in Primary Hepatocytes for the Correction of Inherited Metabolic Liver Disease
by Justin Gibson, Abishek Dhungana, Menam Pokhrel, Benjamin Arthur, Pramita Suresh, Olumide Adebayo and Renee N. Cottle
Cells 2025, 14(10), 711; https://doi.org/10.3390/cells14100711 - 14 May 2025
Viewed by 344
Abstract
Hepatocyte transplantation (HTx) combined with ex vivo gene therapy has garnered significant interest due to its potential for treating many inherited metabolic liver diseases. The biggest obstacle for HTx is achieving sufficient engraftment levels to rescue diseased phenotypes, which becomes more challenging when [...] Read more.
Hepatocyte transplantation (HTx) combined with ex vivo gene therapy has garnered significant interest due to its potential for treating many inherited metabolic liver diseases. The biggest obstacle for HTx is achieving sufficient engraftment levels to rescue diseased phenotypes, which becomes more challenging when combined with ex vivo gene editing techniques. However, recent technological advancements have improved electroporation delivery efficiency, cell viability, and scalability for cell therapy. We recently demonstrated the impacts of electroporation for cell-based gene therapy in a mouse model of hereditary tyrosinemia type 1 (HT1). Here, we explore the use of the clinical-grade electroporator, the MaxCyte ExPERT GTx, utilized in the first FDA-approved CRISPR therapy, Casgevy, and evaluate its potential in primary hepatocytes in terms of delivery efficiency and cell viability. We assessed the gene editing efficiency and post-transplantation engraftment of hepatocytes from mTmG mice electroporated with CRISPR-Cas9-ribonucleoproteins (RNPs) targeting 4-hydroxyphenylpyruvate dioxygenase (Hpd) in a fumarylacetoacetate hydrolase (Fah)-deficient mouse model of HT1. After surgery, Fah-/- graft recipients were cycled off and on nitisinone to achieve independence from drug-induced Hpd inhibition, an indicator of HT1 disease correction. Transplanted hepatocytes subjected to electroporation using the GTx system had a cell viability of 89.9% and 100% on-target gene editing efficiency. Recipients transplanted with GTx-electroporated cells showed a smaller weight reduction than controls transplanted with untransfected cells (7.9% and 13.8%, respectively). Further, there were no mortalities in the GTx-recipient mice, whereas there was 25% mortality in the control recipients. Mean donor cell engraftment was significantly higher in GTx-recipient mice compared to untransfected control recipients (97.9% and 81.6%, respectively). Our results indicate that the GTx system does not negatively impact hepatocyte functionality and engraftment potential, thereby demonstrating the promise of GTx electroporation in hepatocytes as a viable cell therapy for treating genetic diseases that affect the liver. Full article
(This article belongs to the Special Issue CRISPR-Based Genome Editing in Translational Research—Third Edition)
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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 353
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)
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