Modern approaches of gene therapy to the treatment of diabetes mellitus

Modern gene therapy approaches have shown promise in the treatment of diabetes, a chronic disease characterized by persistently high blood glucose levels due to insufficient insulin production, the incidence of which is expected to increase by 11.3% by 2030. This review includes a consideration of modern approaches to gene therapy in the treatment of diabetes, a description of the main technologies with an indication of their shortcomings. CRISPR/Cas9 technology is explained: a system that identifies a target region of the genome and performs a double-stranded DNA cut, after which the required sequence is inserted. Long-term efficacy in achieving normoglycemia without the need for exogenous insulin has been demonstrated using adeno-associated virus vectors. And research with the pVAX plasmid allowed for temporary expression of target genes, and the insulin-like growth factor gene was successfully expressed. However, despite the effectiveness of gene therapy technologies, at the present stage there are a number of restrictions on their use in clinical practice, since there are some doubts about their safety during random integration of the virus into the host genome, as well as shortcomings in transduction efficiency and lack of cellular specificity. Therefore, we can conclude that further scientific research is needed in this area.