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...

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Main Authors:	Rhia Ghosh, Sarah J. Tabrizi
Format:	Article
Language:	English
Published:	BMC 2017-10-01
Series:	Alzheimer’s Research & Therapy
Subjects:	Gene suppression RNA interference Anti-sense oligonucleotides Zinc-finger proteins CRISPR/Cas9 Therapeutics
Online Access:	http://link.springer.com/article/10.1186/s13195-017-0307-1

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spelling	doaj-7f6bbf8b607e4b7d830ae17ed046150c2020-11-25T00:48:54ZengBMCAlzheimer’s Research & Therapy1758-91932017-10-019111310.1186/s13195-017-0307-1Gene suppression approaches to neurodegenerationRhia Ghosh0Sarah J. Tabrizi1UCL Huntington’s Disease Centre, Department of Neurodegenerative Disease, UCL Institute of NeurologyUCL Huntington’s Disease Centre, Department of Neurodegenerative Disease, UCL Institute of NeurologyAbstract 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.http://link.springer.com/article/10.1186/s13195-017-0307-1Gene suppressionRNA interferenceAnti-sense oligonucleotidesZinc-finger proteinsCRISPR/Cas9Therapeutics
collection	DOAJ
language	English
format	Article
sources	DOAJ
author	Rhia Ghosh Sarah J. Tabrizi
spellingShingle	Rhia Ghosh Sarah J. Tabrizi Gene suppression approaches to neurodegeneration Alzheimer’s Research & Therapy Gene suppression RNA interference Anti-sense oligonucleotides Zinc-finger proteins CRISPR/Cas9 Therapeutics
author_facet	Rhia Ghosh Sarah J. Tabrizi
author_sort	Rhia Ghosh
title	Gene suppression approaches to neurodegeneration
title_short	Gene suppression approaches to neurodegeneration
title_full	Gene suppression approaches to neurodegeneration
title_fullStr	Gene suppression approaches to neurodegeneration
title_full_unstemmed	Gene suppression approaches to neurodegeneration
title_sort	gene suppression approaches to neurodegeneration
publisher	BMC
series	Alzheimer’s Research & Therapy
issn	1758-9193
publishDate	2017-10-01
description	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.
topic	Gene suppression RNA interference Anti-sense oligonucleotides Zinc-finger proteins CRISPR/Cas9 Therapeutics
url	http://link.springer.com/article/10.1186/s13195-017-0307-1
work_keys_str_mv	AT rhiaghosh genesuppressionapproachestoneurodegeneration AT sarahjtabrizi genesuppressionapproachestoneurodegeneration
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Gene suppression approaches to neurodegeneration

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