New Avenues for the Treatment of Huntington's Disease

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dc.contributor.authorKim, Amy
dc.contributor.authorLalonde, Kathryn
dc.contributor.authorTruesdell, Aaron
dc.contributor.authorGomes Welter, Priscilla
dc.contributor.authorBrocardo, Patricia S.
dc.contributor.authorRosenstock, Tatiana R.
dc.contributor.authorGil-Mohapel, Joana
dc.date.accessioned2021-08-17T22:55:48Z
dc.date.available2021-08-17T22:55:48Z
dc.date.copyright2021en_US
dc.date.issued2021
dc.description.abstractHuntington’s disease (HD) is a neurodegenerative disorder caused by a CAG expansion in the HD gene. The disease is characterized by neurodegeneration, particularly in the striatum and cortex. The first symptoms usually appear in mid-life and include cognitive deficits and motor disturbances that progress over time. Despite being a genetic disorder with a known cause, several mechanisms are thought to contribute to neurodegeneration in HD, and numerous pre-clinical and clinical studies have been conducted and are currently underway to test the efficacy of therapeutic approaches targeting some of these mechanisms with varying degrees of success. Although current clinical trials may lead to the identification or refinement of treatments that are likely to improve the quality of life of those living with HD, major efforts continue to be invested at the pre-clinical level, with numerous studies testing novel approaches that show promise as disease-modifying strategies. This review offers a detailed overview of the currently approved treatment options for HD and the clinical trials for this neurodegenerative disorder that are underway and concludes by discussing potential disease-modifying treatments that have shown promise in pre-clinical studies, including increasing neurotropic support, modulating autophagy, epigenetic and genetic manipulations, and the use of nanocarriers and stem cells.en_US
dc.description.reviewstatusRevieweden_US
dc.description.scholarlevelFacultyen_US
dc.description.sponsorshipThis research was funded by the University of Victoria (UVic, Victoria, BC, Canada)–São Paulo Research Foundation (FAPESP, São Paulo, SP, Brazil) SPRINT partnership (UVic-FAPESP SPRINT 1/2018 Grant).en_US
dc.identifier.citationKim, A., Lalonde, K., Truesdell, A., Gomes Welter, P., Brocardo, P. S., Rosenstock, T. R., & Gil- Mohapel, J. (2021). New Avenues for the Treatment of Hungtington’s Disease. International Journal of Molecular Sciences, 22(16), 1-50. https://doi.org/10.3390/ijms22168363.en_US
dc.identifier.urihttps://doi.org/10.3390/ijms22168363
dc.identifier.urihttp://hdl.handle.net/1828/13263
dc.language.isoenen_US
dc.publisherInternational Journal of Molecular Sciencesen_US
dc.subjectautophagy
dc.subjectclinical trial
dc.subjectdisease-modifying treatment
dc.subjectepigenetics
dc.subjectgenetics
dc.subjectHuntington's disease
dc.subjectpre-clinical study
dc.subjecttransgenic mouse model
dc.subjecttrophic support
dc.subjectIsland Medical Program
dc.subject.departmentDivision of Medical Sciences
dc.subject.departmentSchool of Medical Sciences
dc.titleNew Avenues for the Treatment of Huntington's Diseaseen_US
dc.typeArticleen_US

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