Advances in Viral Vectors for Gene and Cell Therapy

A special issue of Viruses (ISSN 1999-4915). This special issue belongs to the section "General Virology".

Deadline for manuscript submissions: 31 December 2026 | Viewed by 1190

Special Issue Editor


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Guest Editor
St. Jude Vector Laboratory, St. Jude Children’s Research Hospital, Memphis, TN 38105, USA
Interests: viral vectors design and manufacturing; virus-like particles; gene and cell therapy

Special Issue Information

Dear Colleagues,

Exploiting the innate ability of viruses to enter cells and deliver genetic material has enabled the development of innovative and powerful tools to advance biomedical research, treat numerous monogenic disorders, and engineer immune cells to combat cancer. Despite multiple gene and cell therapy drugs approved by the U.S. Food and Drug Administration and the European Medicines Agency in recent years, many diseases still lack effective therapeutic options. Improvements are needed to enhance the current viral delivery platforms by expanding viral vector cargo capacity, regulating gene expression, achieving targeted cell delivery, advancing manufacturing technologies, and refining safety profiles.

The objective of this Special Issue is to collect high-quality original research manuscripts and review articles that highlight recent advances and improvements in the design, development, and application of viral vectors and virus-like particles for cell and gene therapy.

Topics of interest for this Special Issue include, but are not limited to, the following:

  • Viral vector design.
  • Pseudotyping of viral vectors.
  • Vector manufacturing and purification.
  • Methodology for characterization and quality control of viral vectors.
  • Pre-clinical development of viral vectors.
  • Immune response to viral vector.
  • Virus-Like Particles for the delivery of therapeutic proteins.

Dr. Francesca Ferrara
Guest Editor

Manuscript Submission Information

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Keywords

  • lentiviral vectors
  • adenoviral vectors
  • adeno-associated viral vectors
  • virus-like particles
  • chimeric antigen receptor
  • gene and cell therapy

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Published Papers (1 paper)

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Research

13 pages, 2716 KB  
Article
Development of a Novel Method to Detect AAV Vector Integration
by Junping Zhang, Thao Thi Dang, Tsai-Yu Lin, Xiangping Yu, Danilo Pellin, Jiahe Tian, Olga Simmons, Emma Kou, Kenneth Cornetta and Weidong Xiao
Viruses 2026, 18(3), 315; https://doi.org/10.3390/v18030315 - 3 Mar 2026
Viewed by 926
Abstract
AAV integration has become an important safety consideration in gene therapy. However, accurately determining integration sites remains challenging due to biases introduced by library preparation methods, sequencing technologies, and bioinformatic pipelines. In this study, we developed a PCR-free amplification based on a CRISPR-Cas9 [...] Read more.
AAV integration has become an important safety consideration in gene therapy. However, accurately determining integration sites remains challenging due to biases introduced by library preparation methods, sequencing technologies, and bioinformatic pipelines. In this study, we developed a PCR-free amplification based on a CRISPR-Cas9 cleavage strategy for AAV DNA that overcomes the limitations of PCR amplification imposed by the ITR structure. When combined with long-read nanopore sequencing, this CRISPR-Cas9-based workflow preserves native AAV integration states and enables unbiased detection of integration junctions. We used AAV-transduced HeLa single-cell clones to evaluate the performance of this approach. To confirm integration site identification, AAV integration junctions were also detected using a probe hybridization capture strategy followed by Illumina short-read sequencing. Integration junctions identified by both methods were further confirmed by PCR. The results showed strong consistency between the two approaches in accurately identifying AAV integration sites in each clone. Overall, these findings demonstrate that the CRISPR-Cas9-enabled, PCR-free long-read sequencing workflow provides a promising tool for characterizing AAV integration events. Full article
(This article belongs to the Special Issue Advances in Viral Vectors for Gene and Cell Therapy)
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