Article: article from journal or magazin.
Review (review): journal as complete as possible of one specific subject, written based on exhaustive analyses from published work.
Approches de gene silencing pour le traitement de la maladie de Huntington [Gene silencing approaches for the treatment of Huntington's disease].
Médecine Sciences : M/s
Publication types: English Abstract ; Journal Article ; Review Publication Status: ppublish
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.
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
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