Viral Vectors in Transgenic Research: Prospects for the Treatment of CNS Diseases and Gene Therapy

Abstract

Viral vectors are of great clinical interest due to their high efficiency, which allows them to find practical applications in gene therapy. The review presents current research data on various fish species as potential models for the use of viral vectors. During the study of high-performance adenoviral vectors data on the stable expression of transgenes in the body of Danio rerio and other fish species was obtained. The features of the use of adeno-associated vectors in the study of transneuronal transfer of the glucuronidase protein in the projection neurons of the ventral tegmental area and striatum to treat metabolic insufficiency in case of mucopolysaccharidosis VII are considered. Taking into consideration the wide set of genetically modified Danio rerio strains, ethical aspects, and the acceptability of applying this model in neurogenic studies, it is clear that Danio rerio, as well as other fish species, can be used as an alternative for early preclinical evaluation of the efficiency of viral vectors.