Abstract
Huntington’s disease (HD) is an autosomal dominant hereditary disorder characterized by chorea (excessive, spontaneous, irregularly timed, abrupt movements), disturbed voluntary motor performance, behavioral changes, and dementia. Functional capacity slowly declines as a result of increasing motor and cognitive deficits until the patient becomes bedridden. The course is progressive, with death usually occurring 15–20 years after disease onset. Death is most frequently caused by aspiration pneumonia.
Access this chapter
Tax calculation will be finalised at checkout
Purchases are for personal use only
References
Agrawal N, Pallos J, Slepko N, et al. Identification of combinatorial drug regimens for treatment of Huntington’s disease using Drosophila. Proc Natl Acad Sci U S A 2005;102:3777–81.
Bachoud-Levi A, Gaura V, Brugieres P, et al. Effect of fetal neural transplants in patients with Huntington’s disease 6 years after surgery: a long-term follow-up study. Lancet Neurol 2006;5:303–9.
Bloch J, Bachoud-Lévi AC, Déglon N, et al. Neuroprotective gene therapy for Huntington’s disease, using polymer-encapsulated cells engineered to secrete human ciliary neurotrophic factor: results of a Phase I Study. Human Gene Ther 2004;15:968–75.
Borlongan CV, Skinner SJM, Geaney M, et al. Neuroprotection by encapsulated choroid plexus in a rodent model of Huntington’s disease. Neuroreport 2004;15:2521–25.
Borrell-Pages M, Canals JM, Cordelieres FP, et al. Cysteamine and cysteamine increase in brain levels of BDNF in Huntington disease via HSJ1b and transglutaminase. J Clin Invest 2006;116:1410–24.
Chopra V, Fox JH, Lieberman G, et al. A small-molecule therapeutic lead for Huntington’s disease: preclinical pharmacology and efficacy of C2–8 in the R6/2 transgenic mouse. Proc Natl Acad Sci U S A 2007;104:16685–9.
Dubinsky R, Gray C. CTYE-I-HD: phase I dose finding and tolerability study of cysteamine (Cystagon®) in Huntington’s disease. Mov Disord 2006;21:530–3.
Gu X, Li C, Wei W, et al. pathological cell-cell interactions elicited by a neuropathogenic form of mutant huntingtin contribute to cortical pathogenesis in HD mice. Neuron 2005;46:433–44.
Hauser RA, Furtado S, Cimino CR, et al. Bilateral human fetal striatal transplantation in Huntington’s disease. Neurology 2002;58:687–95.
Hersch SM, Gevorkian S, Marder K, et al. Creatine in Huntington disease is safe, tolerable, bioavailable in brain and reduces serum 8OH2′dG. Neurology 2006;66:250–2.
Huntington Study Group. Dosage effects of riluzole in Huntington’s disease. Neurology 2003;61:1551–6.
Iijima-Ando K, Wu P, Drier EA, et al. cAMP-response element-binding protein and heat-shock protein 70 additively suppress polyglutamine-mediated toxicity in Drosophila. Proc Natl Acad Sci U S A 2005;102:10261–6.
Jin K, LaFevre-Bernt M, Sun Y, et al. FGF-2 promotes neurogenesis and neuroprotection and prolongs survival in a transgenic mouse model of Huntington’s disease. Proc Natl Acad Sci U S A 2005;102:18189–94.
Keene CD, Rodrigues CM, Eich T, et al. Tauroursodeoxycholic acid, a bile acid, is neuroprotective in a transgenic animal model of Huntington’s disease. Proc Natl Acad Sci U S A 2002;90:10671–6.
Kells AP, Fong DM, Dragunow M, et al. AAV-mediated gene delivery of BDNF or GDNF is neuroprotective in a model of Huntington disease. Mol Ther 2004;9:682–8.
Luthi-Carter R, Taylor DM, Pallos J, et al. SIRT2 inhibition achieves neuroprotection by decreasing sterol biosynthesis. Proc Natl Acad Sci U S A 2010;107:7927–32.
McBride JL, Ramaswamy S, Gasmi M, et al. Viral delivery of glial cell line-derived neurotrophic factor improves behavior and protects striatal neurons in a mouse model of Huntington’s disease. Proc Natl Acad Sci U S A 2006;103:9345–50.
Nagai Y, Fujikake N, Ohno K, et al. Prevention of polyglutamine oligomerization and neurodegeneration by the peptide inhibitor QBP1 in Drosophila. Hum Mol Genet 2003;12:1253–9.
Nguyen T, Hamby A, Massa SM. Clioquinol down-regulates mutant huntingtin expression in vitro and mitigates pathology in a Huntington’s disease mouse model. Proc Natl Acad Sci U S A 2005;102:11840–5.
Okamoto S, Pouladi MA, Talantova M, et al. Balance between synaptic versus extrasynaptic NMDA receptor activity influences inclusions and neurotoxicity of mutant huntingtin. Nat Med 2009;15:1407–13.
Patassini S, Giampà C, Martorana A, et al. Effects of simvastatin on neuroprotection and modulation of Bcl-2 and BAX in the rat quinolinic acid model of Huntington’s disease. Neurosci Lett 2008;448:166–9.
Pinto JT, Van Raamsdonk JM, Leavitt BR, et al. Treatment of YAC128 mice and their wild-type littermates with cysteamine does not lead to its accumulation in plasma or brain: implications in the treatment of Huntington disease. Neurochem 2005;94:1087–101.
Puri BK, Leavitt BR, Hayden MR, et al. Ethyl-EPA in Huntington disease: a double-blind, randomized, placebo-controlled trial. Neurology 2005;65:286–92.
Roppongi T, Togo T, Nakamura S, et al. Perospirone in treatment of Huntington’s disease: A first case report. Prog Neuropsychopharmacol Biol Psychiatry 2006;31:308–10.
Sah DW. Therapeutic potential of RNA interference for neurological disorders. Life Sci 2006;79:1773–80.
Sarkar S, Perlstein EO, Imarisio S, et al. Small molecules enhance autophagy and reduce toxicity in Huntington’s disease models. Nat Chem Biol 2007;3:331–8.
Tang TS, Slow E, Lupu V, et al. Disturbed Ca2+ signaling and apoptosis of medium spiny neurons in Huntington’s disease. Proc Natl Acad Sci U S A 2005;102:2602–7.
Tang TS, Chen X, Liu J, Bezprozvanny I. Dopaminergic signaling and striatal neurodegeneration in Huntington’s disease. J Neurosci 2007;27:7899–910.
The Huntington Study Group. A randomized, placebo-controlled trial of coenzyme Q10 and remacemide in Huntington’s disease. Neurology 2001;57:397–404.
Thomas EA, Coppola G, Desplats PA, et al. The HDAC inhibitor 4b ameliorates the disease phenotype and transcriptional abnormalities in Huntington’s disease transgenic mice. Proc Natl Acad Sci U S A 2008;105:15564–9.
Wolfgang WJ, Miller TW, Webster JM, et al. Suppression of Huntington’s disease pathology in Drosophila by human single-chain Fv antibodies. Proc Natl Acad Sci U S A 2005;102:11563–8.
Zhang X, Smith DL, Meriin AB, et al. A potent small molecule inhibits polyglutamine aggregation in Huntington’s disease neurons and suppresses neurodegeneration in vivo. Proc Natl Acad Sci U S A 2005;102:892–97.
Author information
Authors and Affiliations
Corresponding author
Rights and permissions
Copyright information
© 2011 Springer Science+Business Media, LLC
About this chapter
Cite this chapter
Jain, K.K. (2011). Neuroprotection in Huntington’s Disease. In: The Handbook of Neuroprotection. Humana Press, Totowa, NJ. https://doi.org/10.1007/978-1-61779-049-2_9
Download citation
DOI: https://doi.org/10.1007/978-1-61779-049-2_9
Published:
Publisher Name: Humana Press, Totowa, NJ
Print ISBN: 978-1-61779-048-5
Online ISBN: 978-1-61779-049-2
eBook Packages: Biomedical and Life SciencesBiomedical and Life Sciences (R0)