A CAG repeat threshold for therapeutics targeting somatic instability in Huntington's disease

SG Aldous, EJ Smith, C Landles, GF Osborne… - Brain, 2024 - academic.oup.com
The Huntington's disease mutation is a CAG repeat expansion in the huntingtin gene that
results in an expanded polyglutamine tract in the huntingtin protein. The CAG repeat is …

Glutamine missense suppressor transfer RNAs inhibit polyglutamine aggregation

R Tennakoon, TMI Bily, F Hasan, S Syal, A Voigt… - … Therapy Nucleic Acids, 2025 - cell.com
Huntington's disease (HD) is caused by polyglutamine (polyQ) repeat expansions in the
huntingtin gene. HD-causative polyQ alleles lead to protein aggregation, which is a …

Suppression of Huntington's Disease Somatic Instability by Transcriptional Repression and Direct CAG Repeat Binding

EW Mathews, SR Coffey, A Gaertner, J Belgrad… - bioRxiv, 2024 - biorxiv.org
Huntington's disease (HD) arises from a CAG expansion in the huntingtin (HTT) gene
beyond a critical threshold. A major thrust of current HD therapeutic development is lowering …

Using gene or cell therapies to treat Huntington's disease

CS Binda, MJ Lelos, AE Rosser, TH Massey - Handbook of Clinical …, 2024 - Elsevier
Huntington's disease is caused by a CAG repeat expansion in the first exon of the HTT gene,
leading to the production of gain-of-toxic-function mutant huntingtin protein species and …

Positron emission tomography (PET) imaging biomarkers in Huntington's disease

L Everix, S Staelens, D Bertoglio - Biomarkers for Huntington's Disease …, 2023 - Springer
Huntington's disease (HD) is a rare, autosomal, and dominantly inherited
neurodegenerative disease characterized by aggregation of mutated Huntingtin protein …

[HTML][HTML] Chronic cholesterol administration to the brain supports complete and long-lasting cognitive and motor amelioration in Huntington's disease

G Birolini, M Valenza, I Ottonelli, F Talpo… - Pharmacological …, 2023 - Elsevier
Evidence that Huntington's disease (HD) is characterized by impaired cholesterol
biosynthesis in the brain has led to strategies to increase its level in the brain of the rapidly …

[HTML][HTML] Early whole-body mutant huntingtin lowering averts changes in proteins and lipids important for synapse function and white matter maintenance in the …

K Shing, E Sapp, A Boudi, S Liu, C Seeley… - Neurobiology of …, 2023 - Elsevier
Expansion of a triplet repeat tract in exon 1 of the HTT gene causes Huntington's disease
(HD). The mutant HTT protein (mHTT) has numerous aberrant interactions with diverse …

mTOR inhibition in Q175 Huntington's disease model mice facilitates neuronal autophagy and mutant huntingtin clearance

P Stavrides, CN Goulbourne, J Peddy, C Huo, M Rao… - bioRxiv, 2024 - biorxiv.org
Huntington's disease (HD) is caused by expansion of the polyglutamine stretch in huntingtin
protein (HTT) resulting in hallmark aggresomes/inclusion bodies (IBs) composed of mutant …

Intrastriatal delivery of a zinc finger protein targeting the mutant HTT gene obviates lipid phenotypes in zQ175DN HD mice

A Iwanowicz, A Boudi, C Seeley, E Sapp, R Miller… - bioRxiv, 2025 - biorxiv.org
Reducing the burden of mutant Huntingtin (mHTT) protein in brain cells is a strategy for
treating Huntington's disease (HD). However, it is still unclear what pathological changes …

[PDF][PDF] A CAG repeat threshold for therapeutics targeting somatic instability in Huntington's

SG Aldous, EJ Smith, C Landles, GF Osborne… - …, 1784 - ranchobiosciences.com
A CAG repeat threshold for therapeutics targeting somatic instability in Huntington's disease
Page 1 A CAG repeat threshold for therapeutics targeting somatic instability in Huntington’s …