Adeno-associated viral (AAV) vectors are a rapidly emerging gene therapy platform for the
treatment of neurological diseases. In preclinical studies, transgenes encoding therapeutic …

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

RNA interference (RNAi) for spinocerebellar ataxia type 1 can prevent and reverse
behavioral deficits and neuropathological readouts in mouse models, with safety and benefit …

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 …

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

Small noncoding RNAs perform multiple regulatory functions in cells, and their exogenous
mimics are widely used in research and experimental therapies to interfere with target gene …

Spinocerebellar ataxia type 1 (SCA1) is an adult-onset neurodegenerative disease caused
by an expansion of the CAG repeat region of the ATXN1 gene. Currently there are no …

Spinocerebellar ataxia type 1 (SCA1) is an autosomal dominant, late-onset
neurodegenerative disease caused by a polyglutamine (polyQ) expansion in the ataxin-1 …

In the absence of a single preventive or disease-modifying strategy, neurodegenerative
diseases are becoming increasingly prevalent in our ageing population. The mechanisms …

Objective Spinocerebellar ataxia type 1 is an autosomal dominant fatal neurodegenerative
disease caused by a polyglutamine expansion in the coding region of ATXN1. We showed …