Strategies for mutant gene expression silencing in Huntingtonâs disease therapy
Inhibition of huntingtin gene
DOI:
https://doi.org/10.18388/pb.2020_313Abstract
Huntington's disease (HD) is a genetic disease caused by expanded CAG repeat tract in exon 1 of the HTT gene that codes for huntingtin. Since the first symptoms of the disease the average life expectancy is 15-20 years, when the symptoms resulting from neurodegeneration are progressing. Therefore, there is a great demand for an effective HD treatment method. Various therapeutic strategies are being developed based on mechanisms of gene expression silencing, including DNA editing techniques. Here, we present the most important currently tested approaches, with particular emphasis on strategies based on the use of antisense oligonucleotides (ASO), RNA interference (RNAi) technology and CRISPR-Cas9. Currently ongoing clinical trials as well as different pharmacological agents are discussed.
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