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Potential disease-modifying therapies for Huntington's disease: lessons learned and future opportunities
Huntington's disease is the most frequent autosomal dominant neurodegenerative disorder;
however, no disease-modifying interventions are available for patients with this disease. The …
however, no disease-modifying interventions are available for patients with this disease. The …
Huntington disease: new insights into molecular pathogenesis and therapeutic opportunities
Huntington disease (HD) is a neurodegenerative disease caused by CAG repeat expansion
in the huntingtin gene (HTT) and involves a complex web of pathogenic mechanisms …
in the huntingtin gene (HTT) and involves a complex web of pathogenic mechanisms …
Polygenic background modifies penetrance of monogenic variants for tier 1 genomic conditions
Genetic variation can predispose to disease both through (i) monogenic risk variants that
disrupt a physiologic pathway with large effect on disease and (ii) polygenic risk that …
disrupt a physiologic pathway with large effect on disease and (ii) polygenic risk that …
Huntington's disease: a clinical review
Huntington's disease (HD) is a fully penetrant neurodegenerative disease caused by a
dominantly inherited CAG trinucleotide repeat expansion in the huntingtin gene on …
dominantly inherited CAG trinucleotide repeat expansion in the huntingtin gene on …
Unraveling molecular and genetic insights into neurodegenerative diseases: Advances in understanding Alzheimer's, Parkinson's, and Huntington's diseases and …
Neurodegenerative diseases are, according to recent studies, one of the main causes of
disability and death worldwide. Interest in molecular genetics has started to experience …
disability and death worldwide. Interest in molecular genetics has started to experience …
CAG repeat not polyglutamine length determines timing of Huntington's disease onset
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 …
HTT CAG repeat sequence, distinct from the length of huntingtin's polyglutamine segment …
Selective vulnerability of layer 5a corticostriatal neurons in Huntington's disease
The properties of the cell types that are selectively vulnerable in Huntington's disease (HD)
cortex, the nature of somatic CAG expansions of mHTT in these cells, and their importance …
cortex, the nature of somatic CAG expansions of mHTT in these cells, and their importance …
[HTML][HTML] On the wrong DNA track: Molecular mechanisms of repeat-mediated genome instability
Expansions of simple tandem repeats are responsible for almost 50 human diseases, the
majority of which are severe, degenerative, and not currently treatable or preventable. In this …
majority of which are severe, degenerative, and not currently treatable or preventable. In this …
[HTML][HTML] Huntingtin lowering strategies for disease modification in Huntington's disease
SJ Tabrizi, R Ghosh, BR Leavitt - Neuron, 2019 - cell.com
Huntington's disease is caused by an abnormally expanded CAG repeat expansion in the
HTT gene, which confers a predominant toxic gain of function in the mutant huntingtin …
HTT gene, which confers a predominant toxic gain of function in the mutant huntingtin …
Mechanisms of DNA damage‐mediated neurotoxicity in neurodegenerative disease
G Welch, LH Tsai - EMBO reports, 2022 - embopress.org
Neurons are highly susceptible to DNA damage accumulation due to their large energy
requirements, elevated transcriptional activity, and long lifespan. While newer research has …
requirements, elevated transcriptional activity, and long lifespan. While newer research has …