The Role of MicroRNAs in Neurodegeneration: Insights from Huntington's Disease

Abstract

MicroRNA (miRNAs) is a single non-coding strand with a small sequence of approximately 21-25 nucleotides, which could be a biomarker or act as a therapeutic agent for disease. This review explores the dynamic role of miRNAs in Huntington's disease (HD), encompassing their regulatory function, potential as diagnostic biomarker tools, and emerging therapeutic applications. We delved into the dysregulation of specific miRNAs in HD, for instance, downregulated levels of miR-9 and miR-124 and increased levels of miR-155 and miR-196a. These alterations highlight the promise of miRNAs as non-invasive tools for early HD detection and disease progression monitoring. Moving beyond diagnosis, the exciting potential of miRNA-based therapies. By mimicking downregulated miRNAs or inhibiting dysregulated ones, we can potentially restore the balance of mutant target gene expression and modify disease progression. Recent research using engineered miRNAs delivered via an adeno-associated virus (AAV) vector in a transgenic HD minipig model demonstrates encouraging results in reducing mutant HD and improving motor function.

Keywords: Diagnosis; Huntington’s disease; MiRNAs; Therapeutic intervention.