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, Bedri Ranxhi, Kozeta Libohova, Emily D. Shaw, Anna J. Barget, Sokol V. Todi, Henry Lauris Paulson, 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.
Neurobiology of Disease · Nov 26, 2019Peer-reviewedNeuroscience