Gene suppression approaches to neurodegeneration.
Alzheimers Res Ther
; 9(1): 82, 2017 Oct 05.
Article
em En
| MEDLINE
| ID: mdl-28982376
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.
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Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Terapia Genética
/
Doenças Neurodegenerativas
Limite:
Animals
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Humans
Idioma:
En
Ano de publicação:
2017
Tipo de documento:
Article