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CpG-depleted adeno-associated virus (AAV) vectors for improved delivery to skeletal muscle

AAV vectors depleted of the toll-like receptor 9 (TLR9) ligand CpG could improve the success of gene therapy for musculoskeletal diseases. In mice injected intramuscularly with an AAV vector expressing a reporter gene, Tlr9-deficient mice had decreased AAV-associated T cell responses,
T helper type 1 (Th1) cell responses and major histocompatibility complex class II (MHCII) expression compared with wild-type mice. In the animals, intramuscular injection with an AAV vector that lacked CpG motifs triggered less of an immune response than an AAV-reporter vector that contained CpG motifs. Next steps could include testing the CpG-depleted AAV vectors in dogs and nonhuman primates.

SciBX 6(28); doi:10.1038/scibx.2013.740
Published online July 25, 2013

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Faust, S.M. et al. J. Clin. Invest.;
published online June 17, 2013;
Contact: James M. Wilson, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pa.