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


Taken together, our findings suggest that PTC readthrough may be a potential therapeutic strategy for FTLD caused by GRN nonsense mutations.

Authors Frew J, Baradaran-Heravi A, Balgi AD, Wu X, Yan TD, Arns S, Shidmoossavee FS, Tan J, Jaquith JB, Jansen-West KR, Lynn FC, Gao FB, Petrucelli L, Feldman HH, Mackenzie IR, Roberge M, Nygaard HB
Journal Molecular neurodegeneration
Publication Date 2020 Mar 16;15(1):21
PubMed 32178712
PubMed Central PMC7075020
DOI 10.1186/s13024-020-00369-5

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