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Adeno-associated virus (AAV) capsid engineering in liver-directed gene therapy

17 Dec 2020

Abstract excerpt

Introduction: Gene therapy clinical trials with adeno-associated virus (AAV) vectors report impressive clinical efficacy data. Nevertheless, challenges have become apparent, such as the need for high vector doses and the induction of anti-AAV immune responses that cause the loss of vector-transduced hepatocytes. This fostered research focusing on development of next-generation AAV vectors capable of dealing with...

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PMID: 33331201
DOI: 10.1080/14712598.2021.1865303

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