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. You will use human in vitro models, including astrocytes and brain organoids generated from induced pluripotent stem cells (iPSCs) of people with MLC and healthy controls. You will investigate disease
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physiologically relevant human in vitro model of Pelizaeus-Merzbacher-like disease type 1 (PMLD1). You will first test leading AAV capsids developed by Myrtelle Therapeutics Inc. in the in vitro model. You will
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liquid biomarkers for leukodystrophies by integrating multi-omics data from patient plasma and iPSC-derived human disease models. You will investigate disease-related molecular signatures using
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to preclinical laboratory research using advanced disease models, including patient-derived organoids and human xenograft tumour models on chicken eggs. You will also investigate why cancer cells are particularly
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, for example through newborn screening, family screening or genetic testing. These families may face uncertainty about when symptoms will develop and difficult decisions about treatment. Understanding