Genomic and transcriptomic quality control for an autologous iPSC-derived cell therapy for Parkinson's disease.

Genomic and transcriptomic qualification of patient-derived dopaminergic neuron precursors was successfully achieved across multiple donors for Parkinson's disease therapy.

• Why it matters: This work addresses the need for reliable quality control in autologous iPSC-derived cell therapies, which are promising due to immune compatibility and reduced side effects but require rigorous validation.
• What they did: The team performed whole-genome sequencing on fibroblasts, iPSCs, and DANPCs from patients and developed NeuriTest, an RNAseq-based tool to predict cell quality based on animal data, using samples from multiple donors.
• The result: The approach demonstrated reproducible quality control, with preclinical efficacy in rodent models and safety confirmed over a 9-month toxicology study, supporting the potential for personalized Parkinson's disease treatment.