Frontotemporal dementia

Frontotemporal dementia affects specific parts of the brain and shrinkage in these regions changes personality, language and behaviour. The personality change can be dramatic and can make it difficult for family members to cope. We have identified many genes involved and have increased understanding in how it links with motor neuron disease – two devastating diseases that can share a common genetic cause. Our discoveries provide targets needed for the development of new drug treatments and we are currently investigating potential gene therapies to remove toxic mutant genes or add missing genes.

Our major focus for the future is to develop and test gene therapies using antisense oligonucleotides and adeno-associated viral gene vectors which can either knock down toxic mutant genes or supplement deleted genes.

Our key questions:

• How does dysfunctional transport inside the cell (between the nucleus and cytoplasm) initiate neurodegeneration in frontotemporal dementia?
• How do the powerhouses of the cells, mitochondria, contribute to frontotemporal dementia?
• Can gene therapies either knock down toxic mutant genes or supplement deleted genes?

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