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ARTICLE | Translation in Brief

Promoting AAV vectors to safety

Why promoters, other genetic cargo in AAV vectors should be tested for toxicity

April 4, 2019 12:47 PM UTC

Companies looking to make AAVs safer for gene therapies may need to test the vectors' genomic cargo for toxicity, according to a new study from a Harvard and Hong Kong group.

Adeno-associated viral (AAV) vectors are widely used for gene therapies because of their low immunogenicity. High doses of some AAV-based therapies have caused toxicity in animals, leading researchers to assume "that the virus capsid proteins or the contaminants from the prep caused inflammation and toxicity," according to Wenjun Xiong, an assistant professor of biomedical sciences at City University of Hong Kong...

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