Premature termination codon readthrough upregulates progranulin expression and improves lysosomal function in preclinical models of GRN deficiency

Jonathan Frew, Alireza Baradaran-Heravi, Aruna D. Balgi, Xiujuan Wu, Tyler D. Yan, Steve Arns, Fahimeh S. Shidmoossavee, Jason Tan, James B. Jaquith, Karen R. Jansen-West, Francis C. Lynn, Fen Biao Gao, Leonard Petrucelli, Howard H. Feldman, Ian R. MacKenzie, Michel Roberge, Haakon B. Nygaard

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Medicine & Life Sciences