Thursday, July 25, 2013
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adeno-associated virus (AAV) vectors for improved delivery to skeletal muscle
AAV vectors depleted of the
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
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
Published online July 25, 2013
Patent and licensing status
Faust, S.M. et al. J.
published online June 17, 2013;
Contact: James M. Wilson, Perelman School of Medicine at the
University of Pennsylvania, Philadelphia, Pa.
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