The current research paradigm for Huntington's disease is based on participants with overt
clinical phenotypes and does not address its pathophysiology nor the biomarker changes …

Huntington's disease is the most frequent autosomal dominant neurodegenerative disorder;
however, no disease-modifying interventions are available for patients with this disease. The …

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 …

The sequencing of the human genome has allowed the study of the genetic architecture of
common diseases: the number of genomic variants that contribute to risk of disease and …

Brain region-specific degeneration and somatic expansions of the mutant Huntingtin (mHTT)
CAG tract are key features of Huntington's disease (HD). However, the relationships among …

CRISPR enzymes require a defined protospacer adjacent motif (PAM) flanking a guide RNA-
programmed target site, limiting their sequence accessibility for robust genome editing …

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 …

The accumulation of protein inclusions is linked to many neurodegenerative diseases that
typically develop in older individuals, due to a combination of genetic and environmental …

RNA molecules with the expanded CAG repeat (eCAGr) may undergo sol–gel phase
transitions, but the functional impact of RNA gelation is completely unknown. Here, we …

The age at onset of motor symptoms in Huntington's disease (HD) is driven by HTT CAG
repeat length but modified by other genes. In this study, we used exome sequencing of 683 …