基本信息
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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.
Research Interests
Papers共 397 篇Author StatisticsCo-AuthorSimilar Experts
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Acta Neurochirurgicano. 1 (2024): 1-9
crossref(2024)
Annals of clinical and translational neurologyno. 7 (2024): 1775-1786
Robbie Muir, Brendan O'Shea,Raquel Iniesta, Urooba Masood, Cristina Cabassi, Domen Planinc,Emma Hodson-Tole,Emmanuel Drakakis,Martyn Boutelle,Christopher E Shaw,James Bashford
crossref(2024)
crossref(2024)
Zoya Huschtscha,Jessica Silver, Michael Gerhardy,Charles S Urwin, Nathan Kenney, Viet Hung Le,Jackson J Fyfe,Simon A Feros,Andrew C Betik,Christopher S Shaw,Luana C Main,Gavin Abbott,
Sports medicine - openno. 1 (2024): 66-66
Stem cell reportsno. 2 (2024): 187-195
Brain : a journal of neurology (2024)
Frontiers in cellular neuroscience (2023): 1112405
medrxiv(2023)
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