Related Experiment Video

Updated: Jan 28, 2026

Whole-brain Segmentation and Change-point Analysis of Anatomical Brain MRI—Application in Premanifest Huntington's Disease
09:06

Whole-brain Segmentation and Change-point Analysis of Anatomical Brain MRI—Application in Premanifest Huntington's Disease

Published on: June 9, 2018

12.6K

New directions in therapeutics for Huntington disease

Katya T Potkin1,1, Steven G Potkin2,2

  • 1Stony Brook School of Medicine, 101 Nicolls Rd, Stony Brook, NY 11794, USA.

Future Neurology
|February 26, 2019
PubMed

Insights

Huntington disease (HD) is a fatal neurodegenerative disorder caused by a genetic mutation. This review explores emerging therapeutics targeting the underlying pathology of mutant huntingtin protein (mHtt) to combat HD.

Area of Science:

  • Neuroscience
  • Genetics
  • Pharmacology

Background:

  • Huntington disease (HD) is an autosomal dominant neurodegenerative disorder characterized by motor, cognitive, and psychiatric decline.
  • Pathology involves CAG repeat expansion in the huntingtin gene, leading to mutant huntingtin protein (mHtt) production.
  • mHtt causes neurotoxicity, brain atrophy, and aggregate formation, with no approved curative treatments currently available.

Purpose of the Study:

  • To review the latest research on promising therapeutics for Huntington disease.
  • To explore therapeutic strategies targeting the pathological mechanisms of mHtt.

Main Methods:

  • Literature review of recent scientific publications.
  • Analysis of studies focusing on novel therapeutic approaches for HD.

Main Results:

  • Identification of various therapeutic strategies targeting mHtt production, aggregation, and toxicity.
  • Exploration of gene-silencing, protein degradation, and neuroprotective agents.

Conclusions:

  • Emerging therapeutics show promise in addressing the core pathology of Huntington disease.
  • Further research and clinical trials are necessary to develop effective treatments for HD.

Keywords:
ASOHuntington diseaseRNAiVMAT2choreadrug therapiesmHtt

More Related Videos

Microtubule Plus-End Dynamics Visualization in Huntington's Disease Model based on Human Primary Skin Fibroblasts10:38

Microtubule Plus-End Dynamics Visualization in Huntington's Disease Model based on Human Primary Skin Fibroblasts

Published on: January 8, 2022

3.2K
Single Synapse Indicators of Glutamate Release and Uptake in Acute Brain Slices from Normal and Huntington Mice08:27

Single Synapse Indicators of Glutamate Release and Uptake in Acute Brain Slices from Normal and Huntington Mice

Published on: March 11, 2020

6.6K

Related Concept Videos

Direct-Acting Cholinergic Agonists: Therapeutic Uses01:11

Direct-Acting Cholinergic Agonists: Therapeutic Uses

Direct-acting cholinergic agonists have many therapeutic uses in various medical fields. Choline esters, including acetylcholine, have limited clinical utility due to their non-selectivity and short duration of action. Still, acetylcholine and carbachol are applied topically during ophthalmologic surgery to induce miosis. Pilocarpine, a muscarinic and ganglionic stimulator, effectively treats open-angle glaucoma and alleviates xerostomia and dry mouth caused by radiotherapy or Sjögren...
1.7K
Therapeutic Index01:13

Therapeutic Index

The therapeutic index of a drug is a key parameter in pharmacology that quantifies the relative safety of a drug by calculating the ratio between the dose that causes toxicity in half the population (50%) to the dose that proves to be effective for half the population (50%). It provides a spectrum of doses for a particular drug ranging from effective to potentially toxic. To illustrate, consider an anticoagulant agent like warfarin. It possesses a narrow window within its therapeutic index to...
6.7K
Directing Effect of Substituents: meta-Directing Groups01:09

Directing Effect of Substituents: meta-Directing Groups

Substituents on the benzene ring that direct an incoming electrophile to undergo substitution at the meta position are called meta directors. All meta directors either have a positive charge on the atom directly bonded to the ring or a partial positive charge. These groups function by withdrawing electrons from the ring through inductive and resonance effects. Consider the carbocation intermediates formed upon the addition of an electrophile on nitrobenzene at the...
5.9K
Therapeutic Communication01:30

Therapeutic Communication

Communication is a lifelong learning process. Through therapeutic communication, nurses can collect relevant assessment data, provide education and counseling, and interact during nursing interventions. Sending and receiving messages occur through verbal and nonverbal communication techniques and can happen separately or simultaneously.
Verbal communication depends on language or a prescribed way of using words so that people can share information effectively. The critical aspects of verbal...
7.9K
Cholinergic Antagonists: Therapeutic Uses01:26

Cholinergic Antagonists: Therapeutic Uses

Antimuscarinic drugs have various therapeutic applications by inhibiting parasympathetic stimulation in different systems. Here are the key therapeutic uses of antimuscarinics:    
Respiratory Tract: Ipratropium, aclidinium, and tiotropium treat asthma, chronic bronchitis, and chronic obstructive pulmonary disease (COPD). They protect against bronchoconstriction caused by irritants like cigarette smoke, sulfur dioxide, and ozone. They also help reduce nasopharyngeal...
1.4K
Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
1.1K