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Article

Genotoxicity in Mice Following AAV Gene Delivery: A Safety Concern for Human Gene Therapy?

1 Feb 2016

Abstract excerpt

Over the past three decades, a dogma has emerged regarding the uniform safety and nonpathogenicity of adeno-associated virus (AAV), for three excellent reasons. First, natural AAV infections in humans are common and until recently had not been associated with disease. Second, the recombinant AAV (rAAV) genome remains predominantly episomal, greatly reducing the risk of insertional mutagenesis and genotoxicity...

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PMID: 26906613
DOI: 10.1038/mt.2016.17

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