Antisense oligonucleotide therapy: from design to the Huntington disease clinic

ME Rook, AL Southwell - BioDrugs, 2022 - Springer
Huntington disease (HD) is a fatal progressive neurodegenerative disorder caused by an
inherited mutation in the huntingtin (HTT) gene, which encodes mutant HTT protein. Though …

Huntington's disease genetics

RH Myers - NeuroRx, 2004 - Springer
Huntington's disease (HD) is a dominantly transmitted neurodegenerative disorder with wide
variation in onset age but with an average age at onset of 40 years. Children of HD gene …

[HTML][HTML] CAG repeat not polyglutamine length determines timing of Huntington's disease onset

JM Lee, K Correia, J Loupe, KH Kim, D Barker… - Cell, 2019 - cell.com
Variable, glutamine-encoding, CAA interruptions indicate that a property of the uninterrupted
HTT CAG repeat sequence, distinct from the length of huntingtin's polyglutamine segment …

Identification of genetic factors that modify clinical onset of Huntington's disease

JM Lee, VC Wheeler, MJ Chao, JPG Vonsattel… - Cell, 2015 - cell.com
As a Mendelian neurodegenerative disorder, the genetic risk of Huntington's disease (HD) is
conferred entirely by an HTT CAG repeat expansion whose length is the primary …

A new model for prediction of the age of onset and penetrance for Huntington's disease based on CAG length

DR Langbehn, RR Brinkman, D Falush… - Clinical …, 2004 - Wiley Online Library
Huntington's disease (HD) is a neurodegenerative disorder caused by an unstable CAG
repeat. For patients at risk, participating in predictive testing and learning of having CAG …

Venezuelan kindreds reveal that genetic and environmental factors modulate Huntington's disease age of onset

US–Venezuela Collaborative … - Proceedings of the …, 2004 - National Acad Sciences
Huntington's disease (HD) is an autosomal dominant neurodegenerative disease caused by
a triplet (CAG) expansion mutation. The length of the triplet repeat is the most important …

CAG repeat expansion in Huntington disease determines age at onset in a fully dominant fashion

JM Lee, EM Ramos, JH Lee, T Gillis, JS Mysore… - Neurology, 2012 - AAN Enterprises
Objective: Age at onset of diagnostic motor manifestations in Huntington disease (HD) is
strongly correlated with an expanded CAG trinucleotide repeat. The length of the normal …

Looking backward to move forward: early detection of neurodegenerative disorders

ST DeKosky, K Marek - Science, 2003 - science.org
Early detection of neurodegenerative disorders would provide clues to the underlying
pathobiology of these diseases and would enable more effective diagnosis and treatment of …

CAG‐repeat length and the age of onset in Huntington disease (HD): a review and validation study of statistical approaches

DR Langbehn, MR Hayden, JS Paulsen… - American Journal of …, 2010 - Wiley Online Library
Huntington disease (HD) is an inherited neuropsychiatric illness caused by polyglutamine
expansion in the gene for the protein huntingtin (HTT)[Huntington's Disease Collaborative …

[HTML][HTML] Huntington's disease accelerates epigenetic aging of human brain and disrupts DNA methylation levels

S Horvath, P Langfelder, S Kwak, J Aaronson… - Aging (Albany …, 2016 - ncbi.nlm.nih.gov
Age of Huntington's disease (HD) motoric onset is strongly related to the number of CAG
trinucleotide repeats in the huntingtin gene, suggesting that biological tissue age plays an …