NIH R01 · 2024
Mechanisms of Thalamocortical Dysfunction and Social Deficits in FTD due to GRN Mutations
Project Summary/Abstract Loss of function progranulin (GRN) mutations, most of which cause haploinsufficiency, are a major genetic cause of frontotemporal dementia (FTD) with TDP-43 pathology (FTD-TDP). Progranulin-boosting therapies are a promising treatment strategy, but the optimal progranulin-boosting strategy remains unclear. Progranulin has pleiotropic effects and undergoes complex trafficking and processing, so the distribution of progranulin across cell types and cellular compartments may determine the efficacy and safety of progranulin-boosting therapies. Optimal progranulin-boosting therapies would retain progranulin’s neurotrophic and anti- inflammatory effects, with minimal risk…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.