Nucleic Acid-Based Therapy Approaches for Huntington's Disease by Tatyana Vagner, Deborah Young, Alexandre Mouravlev

“…Huntington's disease (HD) is caused by a dominant mutation that results in an unstable expansion of a CAG repeat in the huntingtin gene leading to a toxic gain of function in huntingtin protein which causes massive neurodegeneration mainly in the striatum and clinical symptoms associated with the disease. …”
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The N17 domain of huntingtin as a multifaceted player in Huntington’s disease by Hyunju Cho

“…Huntington’s disease (HD) is primarily caused by the aberrant aggregation of the N-terminal exon 1 fragment of mutant huntingtin protein (mHttex1) with expanded polyglutamine (polyQ) repeats in neurons. …”
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Peripheral huntingtin silencing does not ameliorate central signs of disease in the B6.HttQ111/+ mouse model of Huntington's disease. by Sydney R Coffey, Robert M Bragg, Shawn Minnig, Seth A Ament, Jeffrey P Cantle, Anne Glickenhaus, Daniel Shelnut, José M Carrillo, Dominic D Shuttleworth, Julie-Anne Rodier, Kimihiro Noguchi, C Frank Bennett, Nathan D Price, Holly B Kordasiewicz, Jeffrey B Carroll

“…Peripheral treatment with ASOs led to persistent reduction of huntingtin protein in peripheral organs, including liver (64% knockdown), brown adipose (66% knockdown), and white adipose tissues (71% knockdown). …”
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Integrative determination of atomic structure of mutant huntingtin exon 1 fibrils implicated in Huntington disease by Mahdi Bagherpoor Helabad, Irina Matlahov, Raj Kumar, Jan O. Daldrop, Greeshma Jain, Markus Weingarth, Patrick C. A. van der Wel, Markus S. Miettinen

“…Abstract Neurodegeneration in Huntington’s disease (HD) is accompanied by the aggregation of fragments of the mutant huntingtin protein, a biomarker of disease progression. …”
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Pleiotropic effects of mutant huntingtin on retinopathy in two mouse models of Huntington's disease by Hui Xu, Anakha Ajayan, Ralf Langen, Jeannie Chen

“…Huntington's disease (HD) is caused by the expansion of a CAG repeat, encoding a string of glutamines (polyQ) in the first exon of the huntingtin gene (HTTex1). This mutant huntingtin protein (mHTT) with extended polyQ forms aggregates in cortical and striatal neurons, causing cell damage and death. …”
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