Adeno-associated viruses (AAVs) are the safest and most effective gene delivery vehicles to
drive long-term transgene expression in gene therapy. While animal studies have shown …

Based on engineered or bacterial nucleases, the development of genome editing
technologies has opened up the possibility of directly targeting and modifying genomic …

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 …

Huntington's disease (HD) is a fatal, dominantly inherited neurodegenerative disorder
caused by CAG trinucleotide expansion in exon 1 of the huntingtin (HTT) gene. Since the …

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 …

Gene regulation is a complex and tightly controlled process that defines cell identity, health
and disease, and response to pharmacologic and environmental signals. Recently …

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 …

Huntington's disease (HD) is an autosomal neurodegenerative disease that is characterized
by an excessive number of CAG trinucleotide repeats within the huntingtin gene (HTT). HD …

Huntington disease (HD) is a fatal dominantly inherited neurodegenerative disorder caused
by CAG repeat expansion (> 36 repeats) within the first exon of the huntingtin gene …

Huntington's disease (HD) is a dominantly inherited neurodegenerative disorder caused by
a CAG trinucleotide expansion in the huntingtin gene (HTT), which codes for the pathologic …