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 …

Adeno-associated viral (AAV) vectors are a rapidly emerging gene therapy platform for the
treatment of neurological diseases. In preclinical studies, transgenes encoding therapeutic …

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 …

Huntington's Disease (HD) is a progressive neurodegenerative disorder caused by CAG
trinucleotide repeat expansions in exon 1 of the huntingtin (HTT) gene. The mutant HTT …

Discovered a little over two decades ago, small interfering RNAs (siRNAs) and microRNAs
(miRNAs) are noncoding RNAs with important roles in gene regulation. They have recently …

Regulated cell death (RCD) is a ubiquitous process in living organisms that is essential for
tissue homeostasis or to restore biological balance under stress. Over the decades, various …

Huntington's disease is a late-onset neurodegenerative disease caused by a CAG
trinucleotide repeat in the gene encoding the huntingtin protein. Despite its well-defined …

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 …

Gene therapy is at the cusp of a revolution for treating a large spectrum of CNS disorders by
providing a durable therapeutic protein via a single administration. Adeno-associated virus …

Huntington's disease (HD) is the most common monogenic neurodegenerative disease and
the commonest genetic dementia in the developed world. With autosomal dominant …