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.
Downloads
Downloads
Published
Issue
Section
License
All journal contents are distributed under the Creative Commons Attribution-ShareAlike 4.0 International (CC BY-SA 4.0) license. Everybody may use the content following terms: Attribution — You must give appropriate credit, provide a link to the license, and indicate if changes were made, ShareAlike — If you remix, transform, or build upon the material, you must distribute your contributions under the same license as the original. There are no additional restrictions — You may not apply legal terms or technological measures that legally restrict others from doing anything the license permits.
Copyright for all published papers © stays with the authors.
Copyright for the journal: © Polish Biochemical Society.
