New directions in therapeutics for Huntington disease

Future Neurology
Katya T Potkin, Steven G Potkin

Abstract

Huntington disease (HD) is an autosomal dominantly inherited neurodegenerative disease that affects motor, cognitive and psychiatric functions, and ultimately leads to death. The pathology of the disease is based on an expansion of CAG repeats in exon 1 of the huntingtin gene on chromosome 4, which produces a mutant huntingtin protein (mHtt). This protein is involved in neurotoxicity and brain atrophy, and can form β-sheets and abnormal mHtt aggregates. Currently, there are no approved effective treatments for HD, although tetrabenazine (Xenazine™) and deutetrabenazine (AUSTEDO™) have been approved for treatment of the motor symptom chorea in HD. This literature review aims to address the latest research on promising therapeutics based on influencing the hypothesized pathological mechanisms.

References

Dec 12, 2001·Neurochemistry International·Marcela V KarpujLawrence Steinman
Apr 5, 2002·Trends in Genetics : TIG·David C Rubinsztein
Aug 1, 2002·Proceedings of the National Academy of Sciences of the United States of America·C Dirk KeeneWalter C Low
Feb 11, 2003·Proceedings of the National Academy of Sciences of the United States of America·Emma HocklyGillian P Bates
Mar 12, 2003·Neurology·Raphael M BonelliFranz Reisecker
Jun 19, 2003·Human Molecular Genetics·Elizabeth J SlowMichael R Hayden
Jul 23, 2003·Archives of Neurology·Padraig O'Suilleabhain, Richard B Dewey
Apr 22, 2004·Nature Reviews. Neuroscience·M Flint Beal, Robert J Ferrante
Sep 10, 2004·Journal of Neural Transmission. Supplementum·A BeisterM Gerlach
Oct 16, 2004·International Clinical Psychopharmacology·Raphael M BonelliHans-Peter Kapfhammer
Oct 21, 2004·The Journal of Biological Chemistry·Gabriella GardianM Flint Beal
Apr 1, 2005·The Journal of Neuroscience : the Official Journal of the Society for Neuroscience·Thomas A BlanpiedJon W Johnson
Apr 7, 2005·Proceedings of the National Academy of Sciences of the United States of America·Scott Q HarperBeverly L Davidson
Nov 11, 2006·Neurobiology of Disease·Nigel I WoodA Jennifer Morton
Dec 5, 2006·Proceedings of the National Academy of Sciences of the United States of America·Hoon RyuRobert J Ferrante
Dec 22, 2006·The Journal of Biological Chemistry·Sovan SarkarDavid C Rubinsztein
Apr 4, 2007·Human Molecular Genetics·Edward C StackRobert J Ferrante
Jul 27, 2007·The Journal of Neuroscience : the Official Journal of the Society for Neuroscience·Tie-Shan TangIlya Bezprozvanny
Aug 19, 2007·Annals of Neurology·G Bernhard LandwehrmeyerUNKNOWN European Huntington's Disease Initiative Study Group
Aug 19, 2007·Movement Disorders : Official Journal of the Movement Disorder Society·Penelope HogarthDimitri Krainc
Aug 30, 2007·Neurochemical Research·Bjørnar HasselPiers C Emson
Sep 14, 2007·The World Journal of Biological Psychiatry : the Official Journal of the World Federation of Societies of Biological Psychiatry·Yu-Chih Shen
Sep 27, 2007·CNS Drug Reviews·Pierre Etienne Chabrier, Michel Auguet
Oct 9, 2007·Human Molecular Genetics·Sovan SarkarDavid C Rubinsztein
Oct 11, 2007·Proceedings of the National Academy of Sciences of the United States of America·Vanita ChopraSteven Hersch

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Citations

Jul 20, 2019·Journal of Huntington's Disease·Inês Caldeira BrásTiago Fleming Outeiro
Aug 23, 2019·Neurotherapeutics : the Journal of the American Society for Experimental NeuroTherapeutics·Anna Niewiadomska-Cimicka, Yvon Trottier
Feb 23, 2020·Reviews in the Neurosciences·Magdalena JabłońskaPrzemysław Gałązka
May 30, 2020·International Journal of Molecular Sciences·Serena StangaAlessandro Vercelli
Jul 18, 2019·Frontiers in Pharmacology·Selene IngusciMichele Simonato
May 20, 2020·Scientific Reports·Claudio Rodríguez-GonzálezChristian Hansen
Aug 28, 2021·International Journal of Molecular Sciences·Amy KimJoana Gil-Mohapel

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Methods Mentioned

BETA
antisense oligonucleotides
histone acetylation
acetylation

Clinical Trials Mentioned

NCT01458470
NCT02231580
NCT01357681
NCT02453061
NCT00497159
NCT01085266
NCT00920946
NCT00387270
NCT00988624
NCT00827034

Software Mentioned

WAVE Life Sciences

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