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

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.

Molecular Neurodegeneration, 2020

Summary:

Learn how Maestro MEA was used to explore restoring GRN deficiency in hiPSC-derived neurons as a potential therapeutic intervention for FTLD.

Keywords: Dementia, Astrocytes, induced pluripotent stem cell