In particular, for a number of disorders classified as lysosomal storage diseases [40], clinical trials were conducted using viral vectors [41], such as Canavan disease [42,43]—a pediatric neurodegenerative disease associated with pathogenic variants in the aspartoacylase gene (ASPA); late infantile neuronal ceroid lipofuscinosis (LINCL) [44]; and a number of other diseases; a detailed analysis of these materials can be found in a number of publications [45,46,47,48]. The gene discussed is ASPA; the disease is lysosomal storage disease.