Chapter 5

The Development of Drug Therapies for Frontotemporal Dementia Caused by Progranulin Mutations

Louis De Muynck and Philip Van Damme


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.

Total Pages: 231-291 (61)

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.Neurodegenerative Diseases: Multifactorial Degenerative Processes, Biomarkers and Therapeutic Approaches.
.Frontiers in Clinical Drug Research - CNS and Neurological Disorders.
.Animal Models for Neurological Disorders.
.Frontiers in Clinical Drug Research – Dementia.
.Recent Advances in the Treatment of Neurodegenerative Disorders.
.Advances in Alzheimer Research.
.Cellular and Molecular Biology of Autism Spectrum Disorders.