Virology and Immunology of Gene Therapy 2025

A special issue of Viruses (ISSN 1999-4915). This special issue belongs to the section "Viral Immunology, Vaccines, and Antivirals".

Deadline for manuscript submissions: 31 December 2025 | Viewed by 243

Special Issue Editors


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Guest Editor
Herman B Wells Center for Pediatric Research, Indiana University, Indianapolis, IN 46202, USA
Interests: gene therapy; AAV; tissue tropism; vector; hemophilia
Special Issues, Collections and Topics in MDPI journals

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Guest Editor
Department of Microbiology, Molecular Genetics and Immunology, University of Kansas Medical Center, Kansas City, KS 66160, USA
Interests: parvovirus; DNA replication; mRNA processing; antiviral; virus receptor; gene therapy
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

The interplay between virology, immunology, and gene therapy is a dynamic and critical area of research. Virology provides the tools and vectors for gene therapy, immunology ensures these tools are safely and effectively accepted by the body, and gene therapy offers new ways to treat diseases by harnessing and modifying these biological systems. Therefore, we have created a Special Issue to address the most important scientific areas: 

  • Development of efficient viral vectors; 
  • Development of new vector production technology to reduce potential immune responses;
  • Better vector characterization technology; 
  • Immunogenicity of viral vectors; 
  • Immunotherapy using gene therapy; 
  • Overcoming pre-existing immunity;  
  • Gene editing for immune modulation; 
  • Autoimmune diseases and gene therapy; 
  • Viral vector-based vaccines.

Prof. Dr. Weidong Xiao
Prof. Dr. Jianming Qiu
Guest Editors

Manuscript Submission Information

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Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2600 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

  • gene therapy
  • viral vector
  • vaccine
  • immune responses
  • viruses
  • AAV
  • lentiviral vectors
  • neutralizing antibody

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Published Papers (1 paper)

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Research

20 pages, 2817 KiB  
Article
A Versatile Reporter Platform for Evaluating HDR- and NHEJ-Based Genome Editing in Airway Epithelial Cell Cultures Using an rAAV Vector
by Soo Yeun Park, Zehua Feng, Xiujuan Zhang, Yinghua Tang, Donovan Richart, Kai E. Vorhies, Jianming Qiu, John F. Engelhardt and Ziying Yan
Viruses 2025, 17(6), 821; https://doi.org/10.3390/v17060821 - 6 Jun 2025
Abstract
Therapeutic gene editing strategies utilize endogenous DNA repair pathways—nonhomologous end joining (NHEJ) or homology-directed repair (HDR)—to introduce targeted genomic modifications. Because HDR is restricted to dividing cells, whereas NHEJ functions in both dividing and non-dividing cells, NHEJ-based approaches are better suited for in [...] Read more.
Therapeutic gene editing strategies utilize endogenous DNA repair pathways—nonhomologous end joining (NHEJ) or homology-directed repair (HDR)—to introduce targeted genomic modifications. Because HDR is restricted to dividing cells, whereas NHEJ functions in both dividing and non-dividing cells, NHEJ-based approaches are better suited for in vivo gene editing in the largely post-mitotic airway epithelium. Homology-independent targeted insertion (HITI), an NHEJ-based method, offers a promising strategy for cystic fibrosis (CF) gene therapy. Here, we applied HITI to drive the expression of a promoterless reporter through an exon trap strategy in both proliferating airway basal cells and well-differentiated primary airway epithelial cultures derived from transgenic ROSAmTmG ferrets. We also established a versatile human gene editing reporter (GER) airway basal cell line capable of multipotent differentiation, enabling real-time visualization of editing outcomes and the quantitative assessment of HDR- and NHEJ-based editing efficiencies. Together, these platforms provide easily accessible tools for optimizing genome editing strategies in the respiratory epithelium and advancing clinically relevant delivery strategies for CF gene therapy. Full article
(This article belongs to the Special Issue Virology and Immunology of Gene Therapy 2025)
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