Neurobiol Dis 2021 Jun 24;153:105314. Epub 2021 Feb 24.
Centre for Molecular Medicine & Therapeutics, Department of Medical Genetics, University of British Columbia, B.C. Children's Hospital, 950 West 28(th) Avenue, Vancouver, BC V5Z 4H4, Canada; Division of Neurology, Department of Medicine, University of British Columbia Hospital, S 192 - 2211 Wesbrook Mall, Vancouver, BC V6T 2B5, Canada; Center for Brain Health, University of British Columbia, Vancouver, BC V6T 1Z3, Canada. Electronic address:
The granulin protein (also known as, and hereafter referred to as, progranulin) is a secreted glycoprotein that contributes to overall brain health. Heterozygous loss-of-function mutations in the gene encoding the progranulin protein (Granulin Precursor, GRN) are a common cause of familial frontotemporal dementia (FTD). Gene therapy approaches that aim to increase progranulin expression from a single wild-type allele, an area of active investigation for the potential treatment of GRN-dependent FTD, will benefit from the availability of a mouse model that expresses a genomic copy of the human GRN gene. Read More