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will contribute to the development and evaluation of gene therapy approaches for PMLD1. Your work includes: establishing and using isogenic iPSC-derived myelinating organoids as a human in vitro disease
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affecting the white matter of the central nervous system. With the introduction of newborn screening and the first gene therapies, affected children can increasingly be identified and treated before symptom
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: identifying factors associated with parental distress, coping and resilience, drawing on developmental and psychological research. Long-term outcomes: investigating how these factors relate to quality of life
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of newborn screening and the first gene therapies, affected children can increasingly be identified and treated before symptom onset. However, minimally invasive biomarkers are still needed to support early
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This doctoral project focuses on establishing isogenic iPSC-derived myelinating organoids as a physiologically relevant human in vitro model of Pelizaeus-Merzbacher-like disease type 1 (PMLD1). You will first
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, using qualitative research, consensus methods and quantitative validation in an international cohort. Risk and resilience: identifying factors associated with parental distress, coping and resilience
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first use existing datasets to identify bacterial, host and environmental determinants of E. coli carriage, bacterial diarrheal infections, and AMR gene (ARG) carriage in infants. They will then support