Abstract

In this chapter, we discuss the development of therapies for frontotemporal dementia caused by progranulin mutations. Although this is a relatively rare and very specific form of neurodegeneration, the upstream disease cause, being haploinsufficiency of the growth factor progranulin, offers straightforward opportunities for therapy development. Substitution of the progranulin deficiency is likely to counteract the detrimental effects of progranulin haploinsufficiency in patients with frontotemporal dementia and may prevent the manifestation of the disease in presymptomatic mutation carriers. As progranulin has neurotrophic and antiinflammatory properties, therapeutic interventions aimed at augmenting progranulin levels may also become useful in other forms of neurodegeneration.