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Validation of a Göttingen Minipig Gene Therapy Biodistribution Assay for AVGN7 Using Droplet Digital Polymerase Chain ReactionNARA Subscribed
Minipig models are rapidly gaining popularity in gene therapy preclinical studies. This includes biodistribution studies that are required by regulatory agencies, but are poorly described in the literature. This is particularly true for ddPCR assays with enhanced sensitivity over traditional “real-time” PCR. We, therefore, validated a novel duplex ddPCR assay for quantifying AVGN7, an AAV gene therapeutic featuring a codon-opt...
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