New Perspectives of Gene Therapy on Polyglutamine Spinocerebellar Ataxias: From Molecular Targets to Novel Nanovectors
Seven of the most frequent spinocerebellar ataxias (SCAs) are caused by a pathological expansion of a cytosine, adenine and guanine (CAG) trinucleotide repeat located in here exonic regions of unrelated genes, which in turn leads to the synthesis of polyglutamine (polyQ) proteins.PolyQ proteins are prone to aggregate and form intracellular inclusio