ABSTRACT Myotonic dystrophy type 1 (DM1) is the most prevalent form of muscular
dystrophy in adults and yet there are currently no treatment options. Although this disease …

Several inherited neurodegenerative disorders are caused by CAG trinucleotide repeat
expansions, which can be located either in the coding region or in the untranslated region …

The cell nucleus is a major site for polyglutamine (polyQ) toxicity, but the underlying
mechanisms involved have yet been fully elucidated. Here, we report that mutant RNAs that …

Myotonic dystrophy type 1 (DM1) is a complex neuromuscular disease characterized by
skeletal muscle wasting, weakness, and myotonia. DM1 is caused by the accumulation of …

A GGGGCC hexanucleotide repeat expansion in the C9orf72 gene is the most common
genetic cause of amyotrophic lateral sclerosis and frontotemporal dementia …

Glycogen synthase kinase-3β (GSK-3β) has emerged as a critical factor in several pathways
involved in hippocampal neuronal maintenance and function. In Huntington's disease (HD) …

Mutant ribonucleic acid (RNA) molecules can be toxic to the cell, causing human disease
through trans-acting dominant mechanisms. RNA toxicity was first described in myotonic …

Spinocerebellar ataxia type 3 is one of the polyglutamine (polyQ) diseases, which are
caused by a CAG-repeat expansion within the coding region of the associated genes. The …

All cases of Huntington's disease (HD) are caused by mutant huntingtin protein (mhtt), yet
the molecular mechanisms that link mhtt to disease symptoms are not fully elucidated. Given …

We report the development of a new class of nucleic acid ligands that is comprised of Janus
bases and the MPγPNA backbone and is capable of binding rCAG repeats in a sequence …