Polyglutamine spinocerebellar ataxias: emerging therapeutic targets
Sign Up to like & getrecommendations! Published in 2020 at "Expert Opinion on Therapeutic Targets"
DOI: 10.1080/14728222.2020.1827394
Abstract: ABSTRACT Introduction Six of the most frequent dominantly inherited spinocerebellar ataxias (SCAs) worldwide – SCA1, SCA2, SCA3, SCA6, SCA7, and SCA17 – are caused by an expansion of a polyglutamine (polyQ) tract in the corresponding… read more here.
Keywords: targets polyglutamine; emerging therapeutic; polyglutamine spinocerebellar; therapeutic targets ... See more keywords
Current Status of Gene Therapy Research in Polyglutamine Spinocerebellar Ataxias
Sign Up to like & getrecommendations! Published in 2021 at "International Journal of Molecular Sciences"
DOI: 10.3390/ijms22084249
Abstract: 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 region of their causative gene. These neurodegenerative ataxic disorders are… read more here.
Keywords: polyglutamine spinocerebellar; gene therapy; spinocerebellar ataxias; therapy ... See more keywords