Shruthi Nagulapalli

Gene editing treatments for Huntington's disease

Started Nov. 5, 2024

Abstract or project description

Huntington’s Disease (HD) is a hereditary neurodegenerative disorder causing medium spiny neurons (MSNs) to gradually deplete. This disorder is characterized by a mutation in the Huntingtin (HTT) gene leading to a series of nucleotide repeats known as cytosine-adenine-guanine (CAG) expansion. Current treatment options, such as medication to reduce cognitive dysfunction and physical therapy, function solely to alleviate the symptoms of HD. While there is no current cure for HD, gene editing involving CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) offers a prospective approach to counter this disorder. This paper explores current HD treatments as well as their limitations. It further examines the mechanisms of CRISPR-Cas9 mediated therapy, highlighting the risks and benefits of this potential approach. Additionally, this paper analyzes preclinical trial data from HD mouse models treated with gene editing.