Preza, E; Hardy, J; Warner, T; Wray, S; (2016) Induced pluripotent stem cell models of frontotemporal dementia. Neuropathology and Applied Neurobiology , 42 (6) pp. 497-520. 10.1111/nan.12334.

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Abstract

The increasing prevalence of dementia in the ageing population combined with the lack of treatments and the burden on national healthcare systems globally, make dementia a public health priority. Despite the plethora of important research findings published over the past two decades, the mechanisms underlying dementia are still poorly understood and the progress in pharmacological interventions is limited. Recent advances in cellular reprogramming and genome engineering technologies offer an unprecedented new paradigm in disease modeling. Induced pluripotent stem cells (iPSCs) have enabled the study of patient-derived neurons in vitro, a significant progress in the field of dementia research. The first studies using iPSCs to model dementia have recently emerged, holding promise for elucidating disease pathogenic mechanisms and accelerating drug discovery. In this review, we summarise the major findings of iPSC-based studies in Frontotemporal Dementia (FTD) and FTD overlapping with Amyotrophic Lateral Sclerosis (FTD/ALS). We also discuss some of the main challenges in the use of iPSCs to model complex, late-onset neurodegenerative diseases such as dementias.

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