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Ziegler, Tilman; Ishikawa, Kiyotake; Hinkel, Rabea; Kupatt, Christian (2018): Translational Aspects of Adeno-Associated Virus-Mediated Cardiac Gene Therapy. In: Human Gene Therapy, Vol. 29, No. 12: pp. 1341-1351
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Abstract

Recombinant adeno-associated virus vectors (rAAVs) represent a reliable tool for basic and translational research, while rAAVs are also making strides in early clinical trials as vehicles for gene transfer. Their low immunogenicity, tissue tropism, and relative safety due to their low rate of genomic integration represent key features, making rAAVs promising instruments as vectors for future gene therapy approaches. Specifically, for cardiovascular gene therapy, rAAVs appear superior to other vector systems such as lenti- and adenoviral vectors due to the ease of accomplishing long-term cardiac expression of target genes and the reduced risk of provoking immune responses or triggering malignancies through genomic integration. However, major obstacles remain to be resolved if rAAVs are to achieve their full potential as gene therapy vectors in clinical trials. The main hurdles prohibiting their sustained success are their limited capacity to carry transgenes of larger sizes, the prevalence of neutralizing antibodies in the general population, and their tissue specificity, which leaves room for improvement. This review discusses the properties of rAAV that make them useful tools in experimental studies and the treatment of cardiovascular disease in patients.