Huntington's disease (HD) is an inherited neurodegenerative disorder, caused by a CAG repeat expansion in the HTT gene, leading to toxic gene products. The disease has a high prevalence for an inherited disorder, affecting approximately 700,000 people worldwide (one in 10,000). Suffering is high for both patients and carers, with death generally occurring within 10 - 20 years of diagnosis. In this study, we will repress the leading therapeutic target in HD (mutant HTT) using cutting-edge imaging to observe the consequences in the CNS. We will use a synthetic designed zinc finger (ZF) against mutant HTT to attack HD pathology at its source (1,2). We will use our established AAV2- and AAV9-based delivery system, while exploring new delivery strategies (Isalan). The efficacy will be measured by biochemical (metabolomic, transcriptomic) and phenotypic effects, especially ex vivo 2 photon whole-brain imaging in mice (3,4) (Brickley). Overall, we aim to develop a practical therapeutic strategy that is based on gene therapy, which will lead to clinical applications in HD, as well as providing detailed imaging data on HD development.
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博士
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0
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any
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申请截止时间：
12月30日
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背景偏好：You should have obtained a Masters degree at distinction level in a relevant area.
招生人：Prof. Mark Isalan
招生邮箱：m.isalan@imperial.ac.uk
招生网页：http://www.imperial.ac.uk/people/m.isalan
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