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Article

Strategy to detect pre-existing immunity to AAV gene therapy

6 Nov 2017

Abstract excerpt

Gene therapy may offer a new treatment option, particularly for patients with severe hemophilia, based on recent research. However, individuals with pre-existing immunity to adeno-associated viruses (AAVs) may be less likely to benefit from AAV vector-based therapies. To study pre-existing AAV5 immunity in humans, we validated two complementary, sensitive, and scalable in vitro assays to detect AAV5 total...

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PMID: 29106404
DOI: 10.1038/gt.2017.95

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