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Updated: Apr 23, 2026

Fractionation for Resolution of Soluble and Insoluble Huntingtin Species
Published on: February 27, 2018
Delivering a disease-modifying treatment for Huntington's disease
Bruno M D C Godinho1, Meenakshi Malhotra2, Caitriona M O'Driscoll2
1Pharmacodelivery Group, School of Pharmacy, University College Cork, Cork, Ireland; Department of Anatomy and Neuroscience, University College Cork, Cork, Ireland.
Huntington's disease (HD) treatments focus on gene-silencing strategies like RNA interference and antisense oligonucleotides to target the toxic mutant Huntingtin gene, showing promise in preclinical studies.
Area of Science:
- Neuroscience
- Genetics
- Molecular Biology
Background:
- Huntington's disease (HD) is a fatal genetic neurodegenerative disorder characterized by motor and cognitive impairments.
- It results from an expanded polyglutamine tract in the Huntingtin (HTT) gene, producing a toxic mutant HTT (muHTT) protein.
Purpose of the Study:
- To review and critically assess the advancements and limitations of various gene-silencing technologies for treating HD.
- To evaluate the therapeutic potential of RNA interference (RNAi), antisense oligonucleotides (ASOs), ribozymes, DNA enzymes, and genome-editing approaches.
Main Methods:
- Literature review of preclinical studies on gene-silencing strategies for HD.
- Critical analysis of the efficacy and challenges associated with each therapeutic approach.
Main Results:
- Several gene-silencing strategies demonstrate significant potential in preclinical models of HD.
- These approaches aim to reduce or repair the expression of the toxic muHTT protein, improving neuropathology and symptoms.
Conclusions:
- Gene-silencing technologies offer promising therapeutic avenues for Huntington's disease, despite current limitations.
- Further research and development are crucial to translate these preclinical findings into effective clinical treatments for HD.
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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
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