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 April 2025) | Viewed by 1816

Special Issue Editors


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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

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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

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Keywords

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

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Published Papers (1 paper)

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Review

15 pages, 1700 KiB  
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 1 | Viewed by 1237
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
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