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Reciprocal mutations of lung-tropic AAV capsids lead to improved transduction properties

22 Nov 2023

Abstract excerpt

Considerable effort has been devoted to developing adeno-associated virus (AAV)-based vectors for gene therapy in cystic fibrosis (CF). As a result of directed evolution and capsid shuffling technology, AAV capsids are available with widespread tropism for airway epithelial cells. For example, AAV2.5T and AAV6.2 are two evolved capsids with improved airway epithelial cell transduction properties over their...

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PMID: 38077224
DOI: 10.3389/fgeed.2023.1271813

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