Zwi-Dantsis, Limor;
Mohamed, Saira;
Massaro, Giulia;
Moeendarbary, Emad;
(2025)
Adeno-Associated Virus Vectors: Principles, Practices, and Prospects in Gene Therapy.
Viruses
, 17
(2)
, Article 239. 10.3390/v17020239.
Preview |
Text
viruses-17-00239.pdf - Published Version Download (2MB) | Preview |
Abstract
Gene therapy offers promising potential as an efficacious and long-lasting therapeutic option for genetic conditions, by correcting defective mutations using engineered vectors to deliver genetic material to host cells. Among these vectors, adeno-associated viruses (AAVs) stand out for their efficiency, versatility, and safety, making them one of the leading platforms in gene therapy. The enormous potential of AAVs has been demonstrated through their use in over 225 clinical trials and the FDA’s approval of six AAV-based gene therapy products, positioning these vectors at the forefront of the field. This review highlights the evolution and current applications of AAVs in gene therapy, focusing on their clinical successes, ongoing developments, and the manufacturing processes required for the rapid commercial growth anticipated in the AAV therapy market. It also discusses the broader implications of these advancements for future therapeutic strategies targeting more complex and multi-systemic conditions and biological processes such as aging. Finally, we explore some of the major challenges currently confronting the field.
| Type: | Article |
|---|---|
| Title: | Adeno-Associated Virus Vectors: Principles, Practices, and Prospects in Gene Therapy |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.3390/v17020239 |
| Publisher version: | https://doi.org/10.3390/v17020239 |
| Language: | English |
| Additional information: | Copyright © 2025 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/). |
| Keywords: | adeno-associated virus (AAV); gene therapy; vector manufacturing; clinical trials |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > UCL BEAMS UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences > UCL School of Pharmacy UCL > Provost and Vice Provost Offices > UCL BEAMS > Faculty of Engineering Science > Dept of Mechanical Engineering UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences > UCL School of Pharmacy > Pharmacology |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10205440 |
Archive Staff Only
 |
View Item |
