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- W4378678889 abstract "Recent progress has led to nucleotide-based gene silencing strategies as a potential therapy for spinocerebellar ataxia type 3 (SCA3) and other hereditary ataxias. But recent setbacks for antisense oligonucleotide (ASO) therapy in another CAG repeat disease, Huntington’s disease (HD), remind us of the importance of broadening the search for potential routes to disease-modifying therapies. Here we review alternative approaches. We begin by introducing some of the complexities and nuances of SCA3 that can help guide therapeutic strategies. We then review the myriad biological pathways that are potentially druggable in SCA3, and the current use of genetic and small molecule screens to identify targets and possible therapeutic agents. Given the importance of employing model systems in which the disease gene and protein are assessed at physiological concentrations in the human genomic context, we discuss the emerging importance and challenges of using human stem cells to study disease mechanisms and test therapeutic targets and novel pharmacologic agents. We conclude by considering SCA3 and related SCAs beyond biochemical pathways and as diseases of impaired connectivity, emphasizing the largely untapped potential of modulating neuronal activity and brain connectivity for possible disease-modifying therapies." @default.
- W4378678889 created "2023-05-30" @default.
- W4378678889 creator A5014931443 @default.
- W4378678889 creator A5055180300 @default.
- W4378678889 creator A5081346285 @default.
- W4378678889 date "2023-01-01" @default.
- W4378678889 modified "2023-09-28" @default.
- W4378678889 title "Seeking Therapies for Spinocerebellar Ataxia: From Gene Silencing to Systems-Based Approaches" @default.
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