New AAV vector targets glomerular endothelial cells

Gene therapy is increasingly used in the clinic, but specific delivery to kidney cells remains an important obstacle to broader implementation in nephrology. In a new study, Tobias Huber and colleagues describe a new adeno-associated virus (AAV) vector that specifically targets glomerular cells.

To test the therapeutic potential of AAV2-GEC, the researchers used it to carry the gene encoding IdeS, the Streptococcus pyogenes protease that cleaves IgG heavy chains. In a mouse model of glomerulonephritis induced by injecting anti-glomerular basement membrane (GBM) sheep serum, treatment with AAV2-GEC-IdeS one day after induction was renoprotective. Treated mice had lower and less persistent albuminuria, and weaker deposition of mouse and sheep IgG on the GBM, compared with control mice injected with AAV2-GEC without IdeS.

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