基本信息
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职业迁徙
个人简介
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 855 篇作者统计合作学者相似作者
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Stem cell reportsno. 2 (2024): 187-195
Frontiers in cellular neuroscience (2023): 1112405
BRAINno. 9 (2023): 3760-3769
medrxiv(2023)
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Research Square (Research Square) (2023)
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Krishnaraj S Rathod,Vikas Kapil,Shanti Velmurugan,Rayomand S Khambata, Umme Siddique,Saima Khan,Sven Van Eijl,Lorna C Gee, Jascharanpreet Bansal, Kavi Pitrola,Christopher Shaw,Fulvio D'Acquisto,
Amyotrophic lateral sclerosis & frontotemporal degenerationpp.1-10, (2023)
Mycological Progressno. 11 (2023): 1-13
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