Related Experiment Video
Updated: Nov 17, 2025

Fractionation for Resolution of Soluble and Insoluble Huntingtin Species
Published on: February 27, 2018
Huntington's Disease: New Frontiers in Therapeutics
1Department of Neurology, The Marlene and Paolo Fresco Institute for Parkinson's and Movement Disorders, NYU Langone Health, 222 East 41st Street - 13th Floor, New York, USA. ling.pan@nyulangone.org.
Purpose Of Review:
This article describes and discusses new potential disease-modifying therapies for Huntington's disease that are currently in human clinical trials as well as promising new therapies in preclinical development.
Recent Findings:
Multiple potential disease-modifying therapeutics for HD are in active development, including direct DNA/gene therapies, RNA modulation, and therapies targeted at aberrant downstream pathways. The etiology of Huntington's disease (HD) is well-known as an abnormally expanded trinucleotide repeat within the huntingtin gene. However, the pathogenesis downstream of the mutant huntingtin gene is complex, involving multiple toxic pathways, including abnormal protein fragmentation and neuroinflammation. The current treatment of HD focuses largely on symptomatic management. This article discusses new, potential disease-modifying therapies that are currently in human clinical trials and preclinical development.
More Related Videos
10:52Efficient and Scalable Production of Full-length Human Huntingtin Variants in Mammalian Cells using a Transient Expression System
Published on: December 10, 2021
11:22Generation of Native, Untagged Huntingtin Exon1 Monomer and Fibrils Using a SUMO Fusion Strategy
Published on: June 27, 2018
Related Concept Videos
Parkinson's Disease: Treatment
Parkinson's Disease is primarily a result of the loss of dopaminergic neurons in the substantia nigra pars compacta. The cornerstone of...
Alzheimer's Disease: Treatment
Drug Discovery: Overview
Targeted Cancer Therapies
There are several types of targeted therapies against...
Parkinson's Disease: Overview
Direct-Acting Cholinergic Agonists: Therapeutic Uses