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Engineered adeno-associated virus 3 vector with reduced reactivity to serum antibodies

29 Apr 2021

Abstract excerpt

The natural serotypes of adeno-associated virus (AAV) or their variants, such as AAV8 and AAV5, are commonly used as vectors in the clinical programs for liver-targeted gene therapy. While AAV8 vectors are not highly efficient at targeting primary human hepatocytes, AAV3 vectors have recently demonstrated remarkable efficiency at targeting both human and non-human primate hepatocytes. However, the presence of...

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PMID: 33927271
DOI: 10.1038/s41598-021-88614-9

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