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TREAT-HD: targeting neurodegeneration in Huntington's disease (360G-Wellcome-200181_Z_15_Z)

Despite their immense public health burden, and after considerable investment in therapeutics research, the pathobiology of neurodegenerative diseases remains poorly understood and we lack treatments to prevent or slow their progression. Our vision is to provide a step-change in the understanding of mechanisms underlying neurodegeneration – and recovery – using Huntington’s Disease (HD) as a model. Our three key goals are to: further understanding of HD neuropathology and its response to gene-silencing treatment. We will exploit a unique opportunity to link with the first human trial of an antisense oligonucleotide (ASO) to reduce levels of huntingtin protein. develop a new generation of ASO treatments by targeting levels of the highly pathogenic exon 1 mutant huntingtin protein. determine the earliest potential time window for therapeutic intervention. We will study a novel cohort of young adult HD gene-carriers decades before expected symptom onset to characterise the earliest signs of disease-related brain changes and identify early functional impairment. By examining this model disease in patients, we will gain understanding of general pathological processes shared across protein-misfolding neurodegenerative disorders such as Alzheimer's and Parkinson's disease. Consequently, this work has fundamental implications for the development of treatment strategies beyond HD to more prevalent neurodegenerative diseases.

£1,693,006

08 Dec 2015

Grant details
Amount Awarded 1693006
Applicant Surname Tabrizi
Approval Committee Science Collaborative Awards Committee
Award Date 2015-12-08T00:00:00+00:00
Financial Year 2015/16
Grant Programme: Title Collaborative Award in Science
Internal ID 200181/Z/15/Z
Lead Applicant Prof Sarah Tabrizi
Other Applicant(s) Prof Barbara Sahakian, Dr Hui Zhang, Prof Geraint Rees, Prof Gillian Bates, Prof Trevor Robbins
Planned Dates: End Date 2021-11-27T00:00:00+00:00
Planned Dates: Start Date 2016-07-01T00:00:00+00:00
Recipient Org: Country United Kingdom
Region Greater London
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