Between cells and selves:The ethics of stem cell research for children with neurodevelopmental disorders

Résumé fourni par la source

Monogenic neurodevelopmental disorders (mNDDs) are genetic conditions characterized by a broad spectrum of somatic, developmental, and psychiatric features. Recent advances in stem cell technology have introduced new opportunities to study these mNDDs. Human somatic cells, such as fibroblasts obtained from skin biopsies and blood cells collected via blood draws, can be reprogrammed into pluripotent stem cells. These induced Pluripotent Stem Cells (iPSCs) can differentiate into diverse cell types and models with the same genetic profile as their donor, such as two-dimensional (2D) neuronal models, which can be used to study neuronal network functioning. iPSC-based neuronal models enable new approaches for disease modeling, drug screening, and treatment development. However, this type of research also raises ethical questions. The main goal of this dissertation is to understand how patient-derived iPSC-based neuronal models can be used ethically in research for children with mNDDs. The research conducted for this dissertation took place within the context of the BRAINmodel project, funded through the PSIDER program of The Netherlands Organisation for Health Research and Development. The BRAINmodel project is a Dutch multidisciplinary consortium in which patient-derived iPSC-based neuronal models are used for disease modeling and developing personalized treatment approaches for children with mNDDs. Three research questions are addressed in this dissertation: 1) What are the experiences and needs of parents of children with rare mNDDs? 2) What is the moral value of patient derived neuronal models? 3) What are the ethical implications of using patient-derived neuronal models in research involving children with mNDDs? These questions are answered by employing a mixed method approach, including both empirical and conceptual research.

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