Approches de gene silencing pour le traitement de la maladie de Huntington [Gene silencing approaches for the treatment of Huntington's disease].
Détails
Demande d'une copie Sous embargo indéterminé.Accès restreint UNIL
Etat: Public
Version: de l'auteur⸱e
Licence: Non spécifiée
ID Serval
serval:BIB_0E4FAB1502A7
Type
Article: article d'un périodique ou d'un magazine.
Sous-type
Synthèse (review): revue aussi complète que possible des connaissances sur un sujet, rédigée à partir de l'analyse exhaustive des travaux publiés.
Collection
Publications
Institution
Titre
Approches de gene silencing pour le traitement de la maladie de Huntington [Gene silencing approaches for the treatment of Huntington's disease].
Périodique
Médecine Sciences : M/s
ISSN
0767-0974 (Print)
ISSN-L
0767-0974
Statut éditorial
Publié
Date de publication
2015
Peer-reviewed
Oui
Volume
31
Numéro
2
Pages
159-167
Langue
français
Notes
Publication types: English Abstract ; Journal Article ; Review Publication Status: ppublish
Résumé
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.
Mots-clé
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
Oui
Création de la notice
01/05/2015 17:17
Dernière modification de la notice
21/01/2021 6:25
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