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National Academy of Sciences, Proceedings of the National Academy of Sciences, 36(112), p. 11276-11281, 2015

DOI: 10.1073/pnas.1503607112

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Genome-wide RNAi screening identifies host restriction factors critical for in vivo AAV transduction

Journal article published in 2015 by Miguel Mano ORCID, Rudy Ippodrino, Lorena Zentilin, Serena Zacchigna, Mauro Giacca
This paper is made freely available by the publisher.
This paper is made freely available by the publisher.

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Abstract

Significance Viral vectors based on the adeno-associated virus (AAV) are at the forefront of human gene therapy; yet, the molecular biology of these vectors is largely unexplored. The aim of this work was to unravel, in a systematic manner, the effect of the individual knockdown of over 18,000 human genes on the efficiency of AAV-mediated transduction. The results obtained connect AAV biology to the processing of cellular DNA damage and pave the way to practical applications to improve therapeutic gene transfer.