Approches de gene silencing pour le traitement de la maladie de Huntington [Gene silencing approaches for the treatment of Huntington's disease].

Details

Serval ID
serval:BIB_0E4FAB1502A7
Type
Article: article from journal or magazin.
Publication sub-type
Review (review): journal as complete as possible of one specific subject, written based on exhaustive analyses from published work.
Collection
Publications
Institution
Title
Approches de gene silencing pour le traitement de la maladie de Huntington [Gene silencing approaches for the treatment of Huntington's disease].
Journal
Médecine Sciences : M/s
Author(s)
Merienne N., Déglon N.
ISSN
0767-0974 (Print)
ISSN-L
0767-0974
Publication state
Published
Issued date
2015
Peer-reviewed
Oui
Volume
31
Number
2
Pages
159-167
Language
french
Notes
Publication types: English Abstract ; Journal Article ; Review Publication Status: ppublish
Abstract
Huntington's disease is a rare neurodegenerative disease caused by a pathologic CAG expansion in the exon 1 of the huntingtin (HTT) gene. Aggregation and abnormal function of the mutant HTT (mHTT) cause motor, cognitive and psychiatric symptoms in patients, which lead to death in 15-20 years. Currently, there is no treatment for HD. Experimental approaches based on drug, cell or gene therapy are developed and reach progressively to the clinic. Among them, mHTT silencing using small non-coding nucleic acids display important physiopathological benefit in HD experimental models.
Keywords
Alleles, Animals, Blood-Brain Barrier, Dependovirus/genetics, Disease Models, Animal, Gene Expression Regulation, Gene Silencing, Genetic Therapy/adverse effects, Genetic Therapy/methods, Genetic Vectors/adverse effects, Genetic Vectors/therapeutic use, Humans, Huntington Disease/genetics, Huntington Disease/therapy, Injections, Intraventricular, Lentivirus/genetics, Mice, Nerve Tissue Proteins/antagonists & inhibitors, Nerve Tissue Proteins/chemistry, Oligonucleotides, Antisense/administration & dosage, Oligonucleotides, Antisense/therapeutic use, Polymorphism, Single Nucleotide, Protein Aggregates, Protein Aggregation, Pathological/etiology, Protein Aggregation, Pathological/genetics, RNA, Messenger/antagonists & inhibitors, RNA, Small Interfering/administration & dosage, RNA, Small Interfering/therapeutic use, Trinucleotide Repeat Expansion/genetics
Pubmed
Web of science
Open Access
Yes
Create date
01/05/2015 17:17
Last modification date
20/08/2019 12:35
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