Gene suppression approaches to neurodegeneration
1UCL Huntington's Disease Centre, Department of Neurodegenerative Disease, UCL Institute of Neurology, London, WC1N 3BG, UK. rhia.ghosh@ucl.ac.uk.
Alzheimer'S Research & Therapy
|October 7, 2017
Summary
Gene suppression therapies like RNA interference and antisense oligonucleotides offer new treatments for neurodegenerative diseases by inhibiting disease-causing proteins. These methods show promise for preventing or reversing disease phenotypes and tackling future proteinopathies.
Area of Science:
- Neuroscience
- Genetics
- Molecular Biology
Background:
- Neurodegenerative diseases pose significant therapeutic challenges.
- Gene suppression strategies have advanced over the past two decades.
- Targeting disease-causing proteins is a key therapeutic goal.
Purpose of the Study:
- To review developments in gene suppression strategies for neurodegenerative diseases.
- To highlight applications in Huntington's disease and inherited neurodegeneration.
- To explore potential for treating other proteinopathy-associated dementias.
Main Methods:
- Review of RNA interference (RNAi) techniques.
- Analysis of anti-sense oligonucleotide (ASO) applications.
- Examination of genome-editing technologies.
Main Results:
- Gene suppression inhibits disease-causing protein expression.
- Post-transcriptional and gene repair mechanisms are employed.
- Successful examples exist in Huntington's disease models.
Conclusions:
- Gene suppression offers a promising therapeutic avenue for neurodegenerative disorders.
- These strategies can potentially prevent or reverse disease phenotypes.
- Future applications may extend to a broader range of proteinopathies.
