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Adeno-associated virus–based gene therapy treats inflammatory kidney disease in mice

15 Aug 2024

Abstract excerpt

Adeno-associated virus (AAV) is a promising in vivo gene delivery platform showing advantages in delivering therapeutic molecules to difficult or undruggable cells. However, natural AAV serotypes have insufficient transduction specificity and efficiency in kidney cells. Here, we developed an evolution-directed selection protocol for renal glomeruli and identified what we believe to be a new vector termed AAV2-GEC...

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PMID: 39225099
DOI: 10.1172/jci174722

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