Gene suppression approaches to neurodegeneration

Abstract Gene suppression approaches have emerged over the last 20 years as a novel therapeutic approach for the treatment of neurodegenerative diseases. These include RNA interference and anti-sense oligonucleotides, both of which act at the post-transcriptional level, and genome-editing techniques...

Full description

Bibliographic Details
Main Authors: Rhia Ghosh, Sarah J. Tabrizi
Format: Article
Language:English
Published: BMC 2017-10-01
Series:Alzheimer’s Research & Therapy
Subjects:
Online Access:http://link.springer.com/article/10.1186/s13195-017-0307-1
Description
Summary:Abstract Gene suppression approaches have emerged over the last 20 years as a novel therapeutic approach for the treatment of neurodegenerative diseases. These include RNA interference and anti-sense oligonucleotides, both of which act at the post-transcriptional level, and genome-editing techniques, which aim to repair the responsible mutant gene. All serve to inhibit the expression of disease-causing proteins, leading to the potential prevention or even reversal of the disease phenotype. In this review we summarise the main developments in gene suppression strategies, using examples from Huntington’s disease and other inherited causes of neurodegeneration, and explore how these might illuminate a path to tackle other proteinopathy-associated dementias in the future.
ISSN:1758-9193