[Gene silencing approaches for the treatment of Huntington's disease].
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.
Lausanne university hospital (CHUV), department of clinical neurosciences (DNC), laboratory of cellular and molecular neurotherapies (LCMN), avenue de Beaumont, Pavillon 3, 1011 Lausanne, Suisse ; Lausanne university hospital (CHUV), neuroscience research center (CRN), LCMN, Lausanne, Suisse.
Nicolas
N
Merienne
Lausanne university hospital (CHUV), department of clinical neurosciences (DNC), laboratory of cellular and molecular neurotherapies (LCMN), avenue de Beaumont, Pavillon 3, 1011 Lausanne, Suisse ; Lausanne university hospital (CHUV), neuroscience research center (CRN), LCMN, Lausanne, Suisse.
Nicole
N
Déglon
D000483:Alleles; D000818:Animals; D001812:Blood-Brain Barrier; D000229:Dependovirus / Q000235:genetics; D004195:Disease Models, Animal; D005786:Gene Expression Regulation; D020868:Gene Silencing; D015316:Genetic Therapy / Q000009:adverse effects / Q000379:methods*; D005822:Genetic Vectors / Q000009:adverse effects / Q000627:therapeutic use; D006801:Humans; D000071058:Huntingtin Protein; D006816:Huntington Disease / Q000235:genetics / Q000628:therapy*; D007276:Injections, Intraventricular; D016086:Lentivirus / Q000235:genetics; D051379:Mice; D009419:Nerve Tissue Proteins / Q000037:antagonists & inhibitors / Q000737:chemistry / Q000235:genetics; D016376:Oligonucleotides, Antisense / Q000008:administration & dosage / Q000627:therapeutic use; D020641:Polymorphism, Single Nucleotide; D066329:Protein Aggregates; D066263:Protein Aggregation, Pathological / Q000209:etiology / Q000235:genetics / Q000517:prevention & control; D012333:RNA, Messenger / Q000037:antagonists & inhibitors; D034741:RNA, Small Interfering / Q000008:administration & dosage / Q000627:therapeutic use; D019680:Trinucleotide Repeat Expansion / Q000235:genetics
C086055:HTT protein, human; D000071058:Huntingtin Protein; D009419:Nerve Tissue Proteins; D016376:Oligonucleotides, Antisense; D066329:Protein Aggregates; D012333:RNA, Messenger; D034741:RNA, Small Interfering