Small Molecule Rescue of ATXN3 Toxicity in C. elegans via TFEB/HLH-30
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Spinocerebellar ataxia type 3 (SCA3), also known as Machado-Joseph disease (MJD), is a polyglutamine expansion disease arising from a trinucleotide CAG repeat expansion in exon 10 of the gene ATXN3. There are no effective pharmacological treatments for MJD, th... ...
