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- W2132281844 abstract "Duchenne muscular dystrophy (DMD) is caused by mutations in the dystrophin gene. Therapeutic gene replacement of a dystrophin cDNA into dystrophic muscle can provide functional dystrophin protein to the tissue. However, vector-mediated gene transfer is limited by anti-vector and anti-transgene host immunity that causes rejection of the therapeutic protein. We hypothesized that rapamycin (RAPA) would diminish immunity due to vector-delivered recombinant dystrophin in the adult mdx mouse model for DMD. To test this hypothesis, we injected limb muscle of mdx mice with RAPA-containing, poly-lactic-co-glycolic acid (PLGA) microparticles prior to dystrophin gene transfer and analyzed treated tissue after 6 weeks. RAPA decreased host immunity against vector-mediated dystrophin protein, as demonstrated by decreased cellular infiltrates and decreased anti-dystrophin antibody production. The interpretation of the effect of RAPA on recombinant dystrophin expression was complex because of an effect of PLGA microparticles." @default.
- W2132281844 created "2016-06-24" @default.
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- W2132281844 date "2012-05-08" @default.
- W2132281844 modified "2023-09-25" @default.
- W2132281844 title "Effect of rapamycin on immunity induced by vector-mediated dystrophin expression in mdx skeletal muscle" @default.
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- W2132281844 doi "https://doi.org/10.1038/srep00399" @default.
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