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Abstract :
[en] expression in fibers localized close to the injection site, while the IVHD caused expression in scattered fibers among the different lower limb muscles.
In conclusion, IVHD and IMEP naked DNA delivery yield different transgene expression levels and patterns which could both be useful in the context of an FSHD model. Indeed, the IMEP leads to a strong and local transgene expression which could allow an easier scoring of AO-mediated DUX4 suppression. The IVHD results in a pattern expected in FSHD patient muscles based on DUX4 stochastic expression observed in primary myoblast and myotube cultures. The IVHD model will thus be preferred for an investigation of pathophysiological mechanisms induced by DUX4 expression.
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