Rescue of progranulin deficiency associated with frontotemporal lobar degeneration by alkalizing reagents and inhibition of vacuolar ATPase.

doi:10.1523/JNEUROSCI.5757-10.2011

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Article

Rescue of progranulin deficiency associated with frontotemporal lobar degeneration by alkalizing reagents and inhibition of vacuolar ATPase.

The Journal of neuroscience : the official journal of the Society for Neuroscience - 2 Feb 2011

Capell Anja, Liebscher Sabine, Fellerer Katrin, Brouwers Nathalie, Willem Michael, Lammich Sven, Gijselinck Ilse, Bittner Tobias, Carlson Aaron M, Sasse Florenz, Kunze Brigitte, Steinmetz Heinrich, Jansen Rolf, Dormann Dorothee, Sleegers Kristel, Cruts Marc, Herms Jochen, Van Broeckhoven Christine, Haass Christian

Abstract excerpt

Numerous loss-of-function mutations in the progranulin (GRN) gene cause frontotemporal lobar degeneration with ubiquitin and TAR-DNA binding protein 43-positive inclusions by reduced production and secretion of GRN. Consistent with the observation that GRN has neurotrophic properties, pharmacological stimulation of GRN production is a promising approach to rescue GRN haploinsufficiency and prevent disease...

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PMID: 21289198
DOI: 10.1523/JNEUROSCI.5757-10.2011
PMCID: PMC6623716

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