Ishani Mallik

Huntington's Disease Research Paper

Started Apr. 25, 2025

Abstract or project description

Huntington’s disease (HD) is an autosomal dominant neurodegenerative disorder. The disease is caused by an expansion of CAG repeats in the huntingtin (HTT) gene, resulting in the production of a toxic huntingtin protein. Characterized by aggregates of mutant huntingtin protein, the disease progressively impairs motor, cognitive, and behavioral functions of the brain. HD pathogenesis involves multiple mechanisms, most notably excitotoxicity, mitochondrial dysfunction, and neuroinflammation. Although the disease has been studied for decades, many current treatments focus on symptom management, rather than slowing or preventing disease progression. As research continues, scientists have developed a new experimental approach—the CRISPR-Cas9 gene-editing system—that holds promise for targeting the underlying genetic cause of the disease. This review examines Huntington’s disease, particularly its pathogenesis, treatments, and experimental approaches for future use.