In Search of Adeno-Associated Virus Vectors With Enhanced Cardiac Tropism for Gene Therapy
Abstract
Globally, adeno-associated virus (AAV) vectors have been increasingly used for clinical gene therapy trials. In Australia, AAV-based gene therapy is available for hereditary diseases such as retinal dystrophy or spinal muscular atrophy 1 (SMA1). Many preclinical studies have used AAV vectors for gene therapy in models of cardiac disease with outcomes of varying translational potential. However, major barriers to effective and safe therapeutic gene delivery to the human heart remain to be overcome. These include tropism, efficient gene transfer, mitigating off-target gene delivery and avoidance of the host immune response. Developing such an enhanced AAV vector for cardiac gene therapy is of great interest to the field of advanced cardiac therapeutics. In this review, we provide an overview of the approaches currently being employed in the search for cardiac cell-specific AAV capsids, ranging from natural AAVs selected as a result of infection and latency in the heart, to the use of cutting-edge molecular techniques to engineer and select AAVs specific for cardiac cells with the use of high-throughput methods.
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BibTeX
@article{sasaki2026search,
title = {{In Search of Adeno-Associated Virus Vectors With Enhanced Cardiac Tropism for Gene Therapy}},
author = {Natsuki Sasaki and Cindy Kok and Adrian Westhaus and Ian Alexander and Leszek Lisowski and Eddy Kizana},
journal = {Heart, Lung and Circulation},
year = {2026},
month = sep,
doi = {10.1016/j.hlc.2023.06.704},
url = {https://hal.science/hal-05772023v1},
note = {CC BY},
}
RIS
TY - JOUR
TI - In Search of Adeno-Associated Virus Vectors With Enhanced Cardiac Tropism for Gene Therapy
AU - Natsuki Sasaki
AU - Cindy Kok
AU - Adrian Westhaus
AU - Ian Alexander
AU - Leszek Lisowski
AU - Eddy Kizana
PY - 2026
DA - 2026/09/30
JO - Heart, Lung and Circulation
DO - 10.1016/j.hlc.2023.06.704
UR - https://hal.science/hal-05772023v1
AB - Globally, adeno-associated virus (AAV) vectors have been increasingly used for clinical gene therapy trials. In Australia, AAV-based gene therapy is available for hereditary diseases such as retinal dystrophy or spinal muscular atrophy 1 (SMA1). Many preclinical studies have used AAV vectors for gene therapy in models of cardiac disease with outcomes of varying translational potential. However, major barriers to effective and safe therapeutic gene delivery to the human heart remain to be overcome. These include tropism, efficient gene transfer, mitigating off-target gene delivery and avoidance of the host immune response. Developing such an enhanced AAV vector for cardiac gene therapy is of great interest to the field of advanced cardiac therapeutics. In this review, we provide an overview of the approaches currently being employed in the search for cardiac cell-specific AAV capsids, ranging from natural AAVs selected as a result of infection and latency in the heart, to the use of cutting-edge molecular techniques to engineer and select AAVs specific for cardiac cells with the use of high-throughput methods.
SN - 1443-9506
SN - 1444-2892
ER -