Author(s) :

Abhinav Donepudi.

Volume/Issue :

Abstract :

Huntington’s disease is a neurological/genetic disorder that affects about 41,000 people in the United States alone. Those with Huntington’s disease live their everyday lives with various symptoms that may greatly hinder their ability to complete specific tasks. Without a cure for Huntington’s disease, people with the disease continue living their lives with these symptoms, including behavioral and movement changes. However, the overexpression of certain microRNAs and artificial microRNAs may prevent neurodegeneration induced by Huntington’s disease. MicroRNAs, a naturally occurring class of non-coding RNA, can regulate the expression of specific proteins by preventing the translation of messenger RNA. Within neurological cells, the overexpression of microRNAs naturally found in the nervous system can promote neurogenesis and neuronal function. This was found to occasionally combat neurodegeneration found in various models of Huntington’s disease, suggesting its therapeutic potential. Artificial microRNAs are genetically engineered microRNAs used to prevent the translation of specific genes. They are different from microRNAs in that they can be engineered to target more particular genes, such as the Huntingtin gene, unlike microRNAs, which usually only target genes that are involved with general cell productivity. By targeting the mutant Huntingtin gene among models of Huntington’s disease, it was consistently found that mutant Huntingtin mRNA and protein levels were reduced, preventing neuronal death. Methods of delivery of these microRNAs and artificial microRNAs include adeno-associated virus (AAV) vectors, lentiviral vectors, and exosomes, where AAV vectors, specifically AAV5, were found to be the most effective as a delivery method.