Article

An integrin-targeting AAV developed using a novel computational rational design methodology presents improved targeting of the skeletal muscle and reduced liver tropism

2023-10-27

Abstract excerpt

<title>Abstract</title> <p>Current adeno-associated virus (AAV) gene therapy using nature-derived AAVs is limited by non-optimal tissue targeting. In the treatment of muscular diseases (MD), high doses are therefore often required, but can lead to severe adverse effects. To lower treatment doses, we rationally designed an AAV that specifically targets skeletal muscle. We employed a novel computational design that...

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Literature Corpus work: 8f2b8f0f-cbf9-5dbe-9053-7413cef67575
DOI: 10.21203/rs.3.rs-3466229/v1

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An integrin-targeting AAV developed using a novel computational rational design methodology presents improved targeting of the skeletal muscle and reduced liver tropismDOI 10.21203/rs.3.rs-3466229/v1Read

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