Loss function

Internal medicine

Phenotype

Biotechnology

Neuroscience

Disease

Penetrant (biochemical)

Dementia

Biology

Gene

Function (biology)

Neurodegeneration

Frontotemporal lobar degeneration

Frontotemporal dementia

Brain function

Cell biology

Genetics

Medicine

Cell

Peripherally delivered progranulin biologic with enhanced CNS biodistribution corrects CNS disease pathology of the GRN mouse model of frontotemporal dementia.

Rescue of a lysosomal storage disorder caused by Grn loss of function with a brain penetrant progranulin biologic