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

BACKGROUND: Frontotemporal lobar degeneration (FTLD) is a devastating and progressive disorder, and a common cause of early onset dementia. Progranulin (PGRN) haploinsufficiency due to autosomal dominant mutations in the progranulin gene (GRN) is an important cause of FTLD (FTLD-GRN), and nearly a q...

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Publicado en:Mol Neurodegener
Autores principales: Frew, Jonathan, Baradaran-Heravi, Alireza, Balgi, Aruna D., Wu, Xiujuan, Yan, Tyler D., Arns, Steve, Shidmoossavee, Fahimeh S., Tan, Jason, Jaquith, James B., Jansen-West, Karen R., Lynn, Francis C., Gao, Fen-Biao, Petrucelli, Leonard, Feldman, Howard H., Mackenzie, Ian R., Roberge, Michel, Nygaard, Haakon B.
Formato: Artigo
Lenguaje:Inglês
Publicado: BioMed Central 2020
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Research Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC7075020/
https://ncbi.nlm.nih.gov/pubmed/32178712
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13024-020-00369-5
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