Mouse Alport podocytes are susceptible to AAV9 transduction in vivo.

ABSTRACT:

Introduction

Alport syndrome features a defective glomerular basement membrane (GBM) due to variants in COL4A3, COL4A4, and COL4A5. The most severe forms, which lack the GBM's collagen α3α4α5(IV) network, progress from hematuria in early childhood to proteinuria, chronic kidney disease, and kidney failure by the age of ∼30. As a monogenic disease without specific treatments, and with podocytes being the only glomerular cells that synthesize collagen α3α4α5(IV), the ability to efficiently deliver genes to Alport podocytes could open up new possibilities for treatment.

Methods

As a proof-of-concept study, we investigated whether podocytes in X-linked Alport mice were susceptible to transduction by adeno-associated virus (AAV)9. ssAAV9-CAG-tdTomato and scAAV9-CMV-Cre were intra

SUBMITTER: Lin MH 

PROVIDER: S-EPMC12831642 | biostudies-literature | 2026 Jan

REPOSITORIES: biostudies-literature