Virology and Immunology of Gene Therapy

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 July 2024 | Viewed by 860

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

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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 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 and immunology and ensures that these tools are safely and effectively accepted by the body, and gene therapy offers new ways to treat diseases by harnessing and modifying biological systems. Therefore, we have created a Special Issue to address the most important scientific areas of the field:

  • 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

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. Viruses is an international peer-reviewed open access monthly 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 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

Published Papers (2 papers)

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Research

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16 pages, 2615 KiB  
Article
Viral Vector Based Immunotherapy for Peanut Allergy
by Miguel Gonzalez-Visiedo, Roland W. Herzog, Maite Munoz-Melero, Sophia A. Blessinger, Joan M. Cook-Mills, Henry Daniell and David M. Markusic
Viruses 2024, 16(7), 1125; https://doi.org/10.3390/v16071125 - 13 Jul 2024
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Abstract
Food allergy (FA) is estimated to impact up to 10% of the population and is a growing health concern. FA results from a failure in the mucosal immune system to establish or maintain immunological tolerance to innocuous dietary antigens, IgE production, and the [...] Read more.
Food allergy (FA) is estimated to impact up to 10% of the population and is a growing health concern. FA results from a failure in the mucosal immune system to establish or maintain immunological tolerance to innocuous dietary antigens, IgE production, and the release of histamine and other mediators upon exposure to a food allergen. Of the different FAs, peanut allergy has the highest incidence of severe allergic responses, including systemic anaphylaxis. Despite the recent FDA approval of peanut oral immunotherapy and other investigational immunotherapies, a loss of protection following cessation of therapy can occur, suggesting that these therapies do not address the underlying immune response driving FA. Our lab has shown that liver-directed gene therapy with an adeno-associated virus (AAV) vector induces transgene product-specific regulatory T cells (Tregs), eradicates pre-existing pathogenic antibodies, and protects against anaphylaxis in several models, including ovalbumin induced FA. In an epicutaneous peanut allergy mouse model, the hepatic AAV co-expression of four peanut antigens Ara h1, Ara h2, Ara h3, and Ara h6 together or the single expression of Ara h3 prevented the development of a peanut allergy. Since FA patients show a reduction in Treg numbers and/or function, we believe our approach may address this unmet need. Full article
(This article belongs to the Special Issue Virology and Immunology of Gene Therapy)
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Review

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16 pages, 2751 KiB  
Review
The Expression and Function of the Small Nonstructural Proteins of Adeno-Associated Viruses (AAVs)
by Cagla Aksu Kuz, Shane McFarlin and Jianming Qiu
Viruses 2024, 16(8), 1215; https://doi.org/10.3390/v16081215 (registering DOI) - 29 Jul 2024
Abstract
Adeno-associated viruses (AAVs) are small, non-enveloped viruses that package a single-stranded (ss)DNA genome of 4.7 kilobases (kb) within their T = 1 icosahedral capsid. AAVs are replication-deficient viruses that require a helper virus to complete their life cycle. Recombinant (r)AAVs have been utilized [...] Read more.
Adeno-associated viruses (AAVs) are small, non-enveloped viruses that package a single-stranded (ss)DNA genome of 4.7 kilobases (kb) within their T = 1 icosahedral capsid. AAVs are replication-deficient viruses that require a helper virus to complete their life cycle. Recombinant (r)AAVs have been utilized as gene delivery vectors for decades in gene therapy applications. So far, six rAAV-based gene medicines have been approved by the US FDA. The 4.7 kb ssDNA genome of AAV encodes nine proteins, including three viral structural/capsid proteins, VP1, VP2, and VP3; four large nonstructural proteins (replication-related proteins), Rep78/68 and Rep52/40; and two small nonstructural proteins. The two nonstructured proteins are viral accessory proteins, namely the assembly associated protein (AAP) and membrane-associated accessory protein (MAAP). Although the accessory proteins are conserved within AAV serotypes, their functions are largely obscure. In this review, we focus on the expression strategy and functional properties of the small nonstructural proteins of AAVs. Full article
(This article belongs to the Special Issue Virology and Immunology of Gene Therapy)
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