Preclinical Characterization of A Recombinant Adeno-Associated Virus Type 1-Pseudotyped Vector Demonstrates Dose-Dependent Injection Site Inflammation And Dissemination of Vector Genomes to Distant Sites

Abstract excerpt

To translate the potential advantages of recombinant adeno-associated virus type 1 (rAAV1) vectors into a clinical application for muscle-directed gene therapy for alpha1 -antitrypsin (AAT) deficiency, we performed safety studies in 170 C57BL/6 mice and 26 New Zealand White rabbits. A mouse toxicology study included 8 cohorts of 10 mice each (5 per sex). Mice were killed either 21 or 90 days after intramuscular...

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