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 disease is devastating to patients and their families—with autosomal dominant
inheritance, onset typically in the prime of adult life, progressive course, and a combination …

Background Huntington's disease is an autosomal-dominant neurodegenerative disease
caused by CAG trinucleotide repeat expansion in HTT, resulting in a mutant huntingtin …

Many late-onset neurodegenerative diseases, including Parkinson's disease and
Huntington's disease, are associated with the formation of intracellular aggregates by toxic …

Delivery of genes to the brain and spinal cord across the blood-brain barrier (BBB) has not
yet been achieved. Here we show that adeno-associated virus (AAV) 9 injected …

In vivo gene replacement for the treatment of inherited disease is one of the most compelling
concepts in modern medicine. Adeno-associated virus (AAV) vectors have been extensively …

The primary cause of Huntington's disease (HD) is expression of huntingtin with a
polyglutamine expansion. Despite an absence of consensus on the mechanism (s) of …

Delivery represents a significant barrier to the clinical advancement of oligonucleotide
therapeutics for the treatment of neurological disorders, such as Huntington's disease …

Causation is everywhere in the world: it features in every science and technology. But how
much do we truly understand it? Do we know what it means to say that one thing is a cause …

Everyone ages, but only some will develop a neurodegenerative disorder in the process.
Disease might occur when cells fail to respond adaptively to age-related increases in …