Biological preparations for medical use in medical and clinical gene therapy and cell therapy; clinical medical reagents for use in gene therapy, gene diagnosis, and gene testing; pharmaceutical preparations, namely, vaccines for use in gene therapy; pharmaceutical preparations for the treatment of Dravet's disease, Alzheimer's disease, ALS, Huntington's disease, Fragile X syndrome, Parkinson's disease, A1AT deficiency, epilepsy, temporal lobe epilepsy, focal cortical dysplasia, Fabry disease, multiple system atrophy, spinocerebellar ataxia and dyslipidemia; gene therapy and prophylaxis products, namely, gene-based biological preparations and medical reagents for use in gene regulation and gene modulation for the treatment of metabolic disorders, ocular disorders, diseases of the nervous system, blood disorders, cancer, inflammatory and auto-immune diseases, vascular disorders, and single gene disorders.