Huntington's disease articles from across Nature Portfolio

The use of CRISPR interference (CRISPRi) to repress the expression of mutant with relative preservation of wild-type huntingtin may represent a potential therapeutic strategy to delay disease progression in a mouse model of Huntington’s disease.

A Cas9-based gene therapy that replaces expanded CAG repeats in the mutant HTT allele causing Huntington’s disease by a normal CAG repeat led to substantial reductions in neurological symptoms in a pig model of the disease.

Huntington’s disease (HD) is a fatal neurodegenerative disorder caused by a genetic mutation in the huntingtin gene (HTT). Here, cryo electron tomography provides insights into the morphology of the cells derived from patients with HD and mouse models of the disease.