Prospects of using gene therapy and nanotechnologies for the treatment of neurodegenerative diseases

Abstract

In the literature review, an attempt is made to systematize scientific literature regarding the possible prospects of applying gene therapy and nanotechnologies for the treatment of neurodegenerative diseases, such as Alzheimer’s disease, Parkinson’s disease, and Huntington’s disease. It provides a brief description of the likely etiology and pathogenesis of these diseases and offers examples of potential gene targets for therapeutic interventions. The review lists the shortcomings of traditional treatment strategies for neurodegenerative diseases, including typical obstacles related to drug delivery pathways, overcoming the blood-brain barrier, toxicity, and more. It describes the prospects of using nanotechnologies as technical tools for modifying various structures at the molecular level to achieve desired characteristics. In particular, nanotechnologies are described as means that have the potential to assist in the diagnosis and treatment of neurodegenerative diseases, facilitate drug delivery to the central nervous system (CNS), and enhance their effectiveness. The possibility of using nanocomplexes, manufactured using various modern technologies, is also considered, including a focus on metallic, inorganic, polymeric, lipid nanoparticles, and more. The review also outlines the prospects of gene therapy, its potential for treating disorders of the nervous system through the delivery of genetic material to produce therapeutic molecules. In the context of gene therapy, special emphasis is placed on CRISPR/Cas9 technology, which has been introduced for gene editing in eukaryotic cells and is considered an economically efficient and promising method. Thus, this literature review is focused on the application of nanotechnologies and gene therapy for the treatment of neurodegenerative diseases.