Huntington's disease is the most frequent autosomal dominant neurodegenerative disorder;
however, no disease-modifying interventions are available for patients with this disease. The …

RNA plays a central role in the expression of all genes. Because any sequence within RNA
can be recognized by complementary base pairing, synthetic oligonucleotides and …

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 …

Antisense oligonucleotides represent a novel therapeutic platform for the discovery of
medicines that have the potential to treat most neurodegenerative diseases. Antisense …

There are no disease-modifying treatments for adult human neurodegenerative diseases.
Here we test RNA-targeted therapies in two mouse models of spinocerebellar ataxia type 2 …

Multiple neurodegenerative diseases are characterized by single-protein dysfunction and
aggregation. Treatment strategies for these diseases have often targeted downstream …

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 …

Antisense oligonucleotides are synthetic single stranded strings of nucleic acids that bind to
RNA and thereby alter or reduce expression of the target RNA. They can not only reduce …

The 25 years since the identification of the gene responsible for Huntington disease (HD)
have stood witness to profound discoveries about the nature of the disease and its …

The development of antisense oligonucleotide therapy is an important advance in the
identification of corrective therapy for neuromuscular diseases, such as spinal muscular …