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 clustered regularly interspaced short palindromic repeats (CRISPR) renaissance was
catalysed by the discovery that RNA-guided prokaryotic CRISPR-associated (Cas) proteins …

Genome editing technologies operate by inducing site-specific DNA perturbations that are
resolved by cellular DNA repair pathways. Products of genome editors include DNA breaks …

Huntington's disease (HD) is a fully penetrant neurodegenerative disease caused by a
dominantly inherited CAG trinucleotide repeat expansion in the huntingtin gene on …

At present, the idea of genome modification has revolutionized the modern therapeutic
research era. Genome modification studies have traveled a long way from gene …

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 …

Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR‐associated
enzyme (Cas) is a naturally occurring genome editing tool adopted from the prokaryotic …

Cas13 nucleases are a class of programmable RNA-targeting CRISPR effector proteins that
are capable of silencing target gene expression in mammalian cells. Here, we demonstrate …

No disease-slowing treatment exists for Huntington's disease, but its monogenic inheritance
makes it an appealing candidate for the development of therapies targeting processes close …