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.