Heterozygous loss-of-function mutations in the GRN gene are a major cause of hereditary frontotemporal dementia, and there is currently no treatment. Here, the authors report and utilize small molecule positive regulators of GRN expression to boost progranulin levels from the remaining functional GRN allele, thus restoring progranulin levels back to normal within the brain.
- Rachel Tesla
- Charlotte Guhl
- Joachim Herz
