The spinocerebellar ataxias (SCAs) comprise more than 40 autosomal dominant
neurodegenerative disorders that present principally with progressive ataxia. Within the past …

Autosomal dominant cerebellar ataxias (ADCAs) are a group of neurodegenerative
disorders characterized by degeneration of the cerebellum and its connections. All ADCAs …

Inherited retinal degenerations (IRDs) represent a diverse group of progressive, visually
debilitating diseases that can lead to blindness in which mutations in genes that are critical …

RNA interference (RNAi) technology has been used for almost two decades to study gene
functions and in therapeutic approaches. It uses cellular machinery and small, designed …

Gene therapy is, potentially, a powerful tool for treating neurodegenerative diseases such as
amyotrophic lateral sclerosis (ALS), spinal muscular atrophy, Parkinson's disease (PD) and …

RNA-targeting approaches are emerging as viable therapeutics that offer an alternative
method to modulate traditionally 'undrugable'targets. In the case of dominantly inherited …

Recent investigations have defined the pathophysiological basis of many hereditary ataxias
(HAs), including loss-of-function as well as gain-of-function mechanisms at either the RNA or …

Polyglutamine spinocerebellar ataxias (SCAs) comprise a heterogeneous group of six
autosomal dominant ataxias caused by cytosine–adenine–guanine repeat expansions in …

Spinocerebellar ataxia (SCA) is a part of the cerebellar neurodegenerative disease group
that is diverse in genetics and phenotypes. It usually shows autosomal dominant …

Polyglutamine spinocerebellar ataxias (PolyQ SCAs) are a group of 6 rare autosomal
dominant diseases, which arise from an abnormal CAG repeat expansion in the coding …