Year: 2023
Type: review
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.
Source: Heart Lung and Circulation
Authors: Natsuki Sasaki, Cindy Y. Kok, Adrian Westhaus, Ian E. Alexander, Leszek Lisowski, Eddy Kizana
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: hybrid
Estimated APC paid: $2,860
Awards: Preclinical Assessment of Gene Therapy for Ventricular Arrhythmia, Strategies for development of novel adeno-associated viral vectors and production technology: towards national clinical-grade manufacturing capacity, Bioengineering of next-generation adeno-associated viral vectors for gene therapy clinical applications using whole human liver explant preclinical model, Preclinical Assessment of Gene Therapy for Creation of a Biological Pacemaker
OpenAlex ID: W4384024818