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Dichotomies in Ex Vivo and In Vivo Performance of Receptor-Binding Mutants of Adeno-Associated Virus Vectors.

Human gene therapy - 1 Aug 2026

Olarewaju Olaniyi, Bentler Martin, Breuer Jacqueline, Alioglu Fatih, Grabowski Anna, Prosche Aileen, Sami Haider, Ogris Manfred, Büning Hildegard

Abstract excerpt

Receptor engagement plays a key role in the cellular uptake, intracellular trafficking, and overall transduction efficiency of adeno-associated virus (AAV) vectors. Although heparan sulfate proteoglycan (HSPG) and α5ß1 integrin-binding motifs of the AAV serotype 2 (AAV2) capsid were mapped, it has remained incompletely understood how loss of these interactions affects AAV vector performance. Hence, we generated...

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PMID: 42459063
DOI: 10.1177/10430342261465461

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