[Gene silencing approaches for the treatment of Huntington's disease]
Nicolas Merienne1, Nicole Déglon1
1Lausanne 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.
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.
More Related Videos
Related Concept Videos
siRNA - Small Interfering RNAs
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the...
Experimental RNAi
RNA Interference
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
MicroRNAs
Pharmacogenomics: Identification of New Drug Targets


