Gene silencing approaches for the treatment of Huntington's disease
Medecine/Sciences, ISSN: 1958-5381, Vol: 31, Issue: 2, Page: 159-167
2015
- 4Citations
- 34Captures
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Example: if you select the 1-year option for an article published in 2019 and a metric category shows 90%, that means that the article or review is performing better than 90% of the other articles/reviews published in that journal in 2019. If you select the 3-year option for the same article published in 2019 and the metric category shows 90%, that means that the article or review is performing better than 90% of the other articles/reviews published in that journal in 2019, 2018 and 2017.
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Review Description
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.
Bibliographic Details
http://www.scopus.com/inward/record.url?partnerID=HzOxMe3b&scp=84924328555&origin=inward; http://dx.doi.org/10.1051/medsci/20153102012; http://www.ncbi.nlm.nih.gov/pubmed/25744262; http://www.medecinesciences.org/10.1051/medsci/20153102012; https://dx.doi.org/10.1051/medsci/20153102012; https://www.medecinesciences.org/articles/medsci/full_html/2015/02/medsci20153102p159/medsci20153102p159.html
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