AAV Vector-Mediated Modulation of Signaling Pathways in Neurological Disorders: Insights From Cellular, Animal, and...

One of the most efficient and promising viral vectors for the treatment of central nervous system (CNS) and peripheral nervous system (PNS)-related diseases is adeno-associated virals (AAVs), which in recent years, researchers have succeeded in substantially managing and mitigating these disorders by leveraging innovative therapeutic mechanisms at the cellular and molecular levels, which are widely utilized due to their favorable characteristics, including a robust safety profile, the ability to cross the blood-brain barrier, and differential tropisms for distinct types of neuronal cells. Clinically, AAVs have high specificity and have therefore become a dynamic and useful tool for the gene therapy of a variety of neurological diseases. Many researchers around the world have been able to study a variety of neurological diseases in vitro cellular models, in vivo animal models, and human clinical studies using these vectors. Given these characteristics, the overall aim of the present review is to investigate the role of AAV vectors carrying coding and non-coding RNAs in the treatment of a variety of neurological disorders, to study the relationship between these vectors and cellular signaling pathways, and to examine its clinical applications in various neurological diseases.