Recent Advances in Adeno-Associated Virus-Mediated Gene Therapy and Vaccine Development

A special issue of Pharmaceutics (ISSN 1999-4923). This special issue belongs to the section "Gene and Cell Therapy".

Deadline for manuscript submissions: closed (20 October 2025) | Viewed by 4746

Special Issue Editors


E-Mail Website
Guest Editor
Wits/SAMRC Antiviral Gene Therapy Research Unit, School of Pathology, Faculty of Health Sciences, University of the Witwatersrand, Johannesburg 2050, South Africa
Interests: HBV; RNAi; siRNA; shRNA; miRNA; cccDNA

E-Mail Website
Guest Editor
Wits/SAMRC Antiviral Gene Therapy Research Unit, Infectious Diseases and Oncology Research Institute (IDORI), Faculty of Health Sciences, University of the Witwatersrand, Parktown 2193, South Africa
Interests: adeno-associated viral vectors; adenoviral vectors; antiviral gene therapy; antiviral vaccine development; hepatitis B virus; hepatitis B virus infection models

Special Issue Information

Dear Colleagues,

AAVs have become the key to gene therapy development in recent years. Multiple lines of evidence highlight AAVs as the safest gene therapy delivery vehicle, and several AAV-based products have been approved for clinical applications. However, there has also been evidence of toxicities in patients following AAV therapy, suggesting that AAVs are not a perfect fit for all. Because AAVs have been thought to stimulate a weaker immune response, they have been overlooked for vaccine development. However, studies illustrating the potential of AAVs as vaccines are starting to emerge. Whether the inherent long-term transgene expression of AAVs could be an advantage or limit their application in vaccine development is still being explored. This Special Issue aims to collate recent findings with regard to the efficacy, safety, and feasibility of using AAVs as gene therapeutics or vaccines against a variety of communicable and non-communicable diseases.

Prof. Dr. Patrick Arbuthnot
Dr. Betty Maepa
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 250 words) can be sent to the Editorial Office for assessment.

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. Pharmaceutics 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 2900 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

  • AAV
  • gene therapy
  • vaccines
  • infectious diseases
  • communicable diseases
  • genetic disorders
  • non-communicable diseases

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.
  • Reprint: MDPI Books provides the opportunity to republish successful Special Issues in book format, both online and in print.

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

Published Papers (2 papers)

Order results
Result details
Select all
Export citation of selected articles as:

Review

15 pages, 1314 KB  
Review
Improvement of Adeno-Associated Virus (AAV)-Based Technologies by Cell-Penetrating Penta-Peptides (CPP5s)
by Charles W. Guo, Anastasia Diener and Shigemi Matsuyama
Pharmaceutics 2026, 18(3), 395; https://doi.org/10.3390/pharmaceutics18030395 - 22 Mar 2026
Viewed by 876
Abstract
Adeno-associated viruses (AAVs) are a promising gene therapy technology, but major technical challenges remain. One problem is that commonly used AAVs have a low efficiency in penetrating the blood–brain barrier (BBB) and the blood–retina barrier (BRB). Consequently, gene delivery to the nervous system [...] Read more.
Adeno-associated viruses (AAVs) are a promising gene therapy technology, but major technical challenges remain. One problem is that commonly used AAVs have a low efficiency in penetrating the blood–brain barrier (BBB) and the blood–retina barrier (BRB). Consequently, gene delivery to the nervous system has limitations. Another problem is that AAVs induce immune reactions that cause serious side effects. To avoid immune reactions, the AAV dose must be reduced to lower levels that may result in insufficient gene delivery. Researchers have been modifying viral capsid protein sequences and searching for effective peptide sequences to solve these problems. As a result, Cell-Penetrating Penta-Peptides (CPP5s) have been shown to be effective in improving the BBB/BRB penetration of AAVs and suppressing immune reactions against AAVs. CPP5s were originally developed from peptide sequences of the Bax (a pro-apoptotic protein) binding domain of Ku70 (a DNA repair protein) and from negative control cell-penetrating peptides without Bax-binding activity. This article will discuss the background science of CPP5s and future directions of CPP5s for AAV-mediated gene delivery to the nervous system as well as other organs. Full article
Show Figures

Figure 1

15 pages, 1700 KB  
Review
Recent Advances in Designing Adeno-Associated Virus-Based Vaccines Against Viral Infections
by Njabulo Mnyandu, Ridhwaanah Jacobs, Patrick Arbuthnot and Mohube Betty Maepa
Pharmaceutics 2024, 16(11), 1360; https://doi.org/10.3390/pharmaceutics16111360 - 24 Oct 2024
Cited by 3 | Viewed by 2949
Abstract
Over 80% of the world’s deadliest pandemics are caused by viral infections, and vaccination remains the most effective way to prevent these infections from spreading. Since the discovery of the first vaccine over two centuries ago, several vaccine design technologies have been developed. [...] Read more.
Over 80% of the world’s deadliest pandemics are caused by viral infections, and vaccination remains the most effective way to prevent these infections from spreading. Since the discovery of the first vaccine over two centuries ago, several vaccine design technologies have been developed. Next-generation vaccines, based on mRNA and viral vector technologies, have recently emerged as alternatives to traditional vaccines. Adenoviral vector-based vaccines against coronavirus disease 2019 have demonstrated a more sustained antibody response as compared to mRNA vaccines. However, this has not been without complications, with a few cases of severe adverse events identified in vaccinated individuals, and the underlying mechanism is the subject of intense investigation. Adeno-associated viral vectors induce a weaker cellular immune response compared to adenoviral vectors, and it is mainly for this reason that there has been a diminished interest in exploring them as a vaccine platform until recently. This review will discuss recent developments and the potential of adeno-associated viral vectors as anti-viral vaccines. Full article
Show Figures

Figure 1

Back to TopTop