基本信息
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职业迁徙
个人简介
I am a Professor of Neurology & Neurogenetics at the IoPPN and Director of The Maurice Wohl Clinical Neuroscience Institute. I am also the Centre Director at UK DRI at King's. My research group focuses on frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) with two main objectives:
To develop novel AAV-based gene therapy therapeutic approaches. Optimising AAV9 vectors for gene delivery into the CNS from preclinical experiments in cells and rodents up to first-in-human experimental medicine studies.
To map TDP-43 proteostasis in vitro using human iPSc-derived neurons: phenotypic analyses including transcriptomics, proteomics and high-content imaging in cultured motor neurons – including patient-derived iPSC and isogenic lines generated by genome editing with CRISPR/Cas9.
To develop novel AAV-based gene therapy therapeutic approaches. Optimising AAV9 vectors for gene delivery into the CNS from preclinical experiments in cells and rodents up to first-in-human experimental medicine studies.
To map TDP-43 proteostasis in vitro using human iPSc-derived neurons: phenotypic analyses including transcriptomics, proteomics and high-content imaging in cultured motor neurons – including patient-derived iPSC and isogenic lines generated by genome editing with CRISPR/Cas9.
研究兴趣
论文共 76 篇作者统计合作学者相似作者
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Thomas P Spargo,Heather Marriott,Guy P Hunt,Oliver Pain,Renata Kabiljo,Harry Bowles,William Sproviero,Alexandra C Gillett,Isabella Fogh, Project MinE ALS Sequencing Consortium, Peter M. Andersen,Nazli A. Başak,
medRxiv (Cold Spring Harbor Laboratory) (2023)
Human Molecular Genetics (2021): 318-320
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