Abstract

PHARMACOLOGICAL TREATMENTS AND THERAPEUTIC APPROACHES FOR HUNTINGTON'S DISEASE

Neha Deepak Gohil*

.

Abstract

The HTT gene's aberrantly enlarged CAG repeat expansion, which results in a dominantly toxic gain of function in the mutant huntingtin (mHTT) protein, is the etiology of Huntington's disease. While there aren't any disease-modifying treatments on the market right now, proximally targeting the pathophysiology of a disease holds a lot of promise. These include post-transcriptional huntingtin-lowering strategies like RNAi, antisense oligonucleotides, and small-molecule splicing modulators; DNA-targeting strategies like zincfinger proteins, transcription activator-like effector nucleases, and CRISPR/Cas9; and novel approaches to remove the mHTT protein like proteolysistargeting chimeras. The establishment of objective biomarkers of disease and HTT reducing pharmacodynamic results, along with advancements in the delivery and distribution of these drugs, have elevated these possible therapeutics to the forefront of Huntington's disease research, with clinical trials underway.

Keywords: .