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