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Understanding AAV vector immunogenicity: from particle to patient

1 Jan 2024

Abstract excerpt

Gene therapy holds promise for patients with inherited monogenic disorders, cancer, and rare genetic diseases. Naturally occurring adeno-associated virus (AAV) offers a well-suited vehicle for clinical gene transfer due to its lack of significant clinical pathogenicity and amenability to be engineered to deliver therapeutic transgenes in a variety of cell types for long-term sustained expression. AAV has been...

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PMID: 38323309
DOI: 10.7150/thno.89380

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