Article
AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington's disease.
Gene therapy - 1 Oct 2017
Miniarikova J, Zimmer V, Martier R, Brouwers C C, Pythoud C, Richetin K, Rey M, Lubelski J, Evers M M, van Deventer S J, Petry H, Déglon N, Konstantinova P
Abstract excerpt
Huntington's disease (HD) is a fatal progressive neurodegenerative disorder caused by a mutation in the huntingtin (HTT) gene. To date, there is no treatment to halt or reverse the course of HD. Lowering of either total or only the mutant HTT expression is expected to have therapeutic benefit. This can be achieved by engineered micro (mi)RNAs targeting HTT transcripts and delivered by an adeno-associated viral...
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