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Gene therapy for the CNS using AAVs: the impact of systemic delivery by AAV9

Each summary identifies the experimental model used

BPCDX, fabricated in a good manufacturing practices (GMP)-certified clean room according to stringent quality processes, was tested to evaluate optical and mechanical properties, enzymatic degradation and cell compatibility and underwent a panel of third-party certified medical device tests compliant with ISO standards to assess biocompatibility, toxicity, carcinogenicity, sensitization and irritation using in vitro and in vivo assays in mice, guinea pigs and rabbits