Genetically Modified MSCs for Targeted Regeneration: Balancing Efficacy, Biosafety, and GMP Standardization
Development of safe-harbor integration and B2M/CIITA-based hypoimmunogenic designs as strategies to reduce engineering-related batch variability and HLA-dependent donor variability support a transition from empirical optimized MSC preparations toward molecularly defined cellular medicines with predefined genotype, expression, potency, and safety attributes.