GENE THERAPY

Safety questions for AAV gene therapy

A long-term study of AAV therapy in dogs finds clonal expansions and vector integrations.

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Fig. 1: Vectors, results and integration analyses for AAV F8 gene therapy.

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Correspondence to Charles P. Venditti.

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Competing interests

C.P.V. is a named inventor on several patents that have been filed by the NIH on his behalf, and his laboratory has received funding from Moderna, Selecta Biosciences, AskBio and LogicBio Therapeutics to support preclinical research.

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Venditti, C.P. Safety questions for AAV gene therapy. Nat Biotechnol (2020). https://doi.org/10.1038/s41587-020-00756-9

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