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Restoring the natural tropism of AAV2 vectors for human liver

9 Sept 2020

Abstract excerpt

(FRG) mice with humanized livers, showing that AAV2 functions poorly in this xenograft model. Here, we derived naturally hepatotropic AAV capsid sequences from primary human liver samples. We demonstrated that capsid mutations, likely acquired as an unintentional consequence of tissue culture propagation, attenuated the intrinsic human hepatic tropism of natural AAV2 and related human liver AAV isolates. These...

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PMID: 32908003
DOI: 10.1126/scitranslmed.aba3312

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