NeuroscienceNeurobiology of DiseasePeer-reviewed
Druggable genome screen identifies new regulators of the abundance and toxicity of ATXN3, the Spinocerebellar Ataxia Type 3 disease protein
Naila S. Ashraf, Joanna R. Sutton, Yemen Yang…·Maria do Carmo Costa
The paper identifies 15 druggable genes that modulate ATXN3 levels and toxicity in SCA3, with FBXL3/SCF pathway showing potential as a therapeutic target.
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