Mingxing Wang

McColl-Lockwood Laboratory for Muscular Dystrophy Research, Carolinas Medical Center, Atrium Health,1000 Blythe Blvd. Charlotte, NC 28203, USA

Publications
  • Research   
    Neutral Surfactant Enhanced Exon-skipping of Morpholino Oligonucleotides in vitro and in mdx Mice
    Author(s): Bo Wu, Drains Morgan, Peijuan Lu, Qilong Lu and Mingxing Wang*

    Antisense oligomers induced exon-skipping has been promising therapy for Duchenne muscular dystrophy in preclinical and clinical trials, but its therapeutic potential could be improved with enhanced delivery approach. A few neutral surfactants were investigated here in for their performance to improve exon-skipping of an antisense phosphorodiamidate morpholino oligomer (PMO) both in vitro and in vivo. This study showed that these surfactants, especially Bigchapand Deoxy Bigchap, improved the delivery efficiency of PMO to the levels comparable to Endoporter-mediated PMO delivery in vitro, and significant enhancement with Deoxy Bigchap-mediated PMO was further observed in mdx mice up to 7-fold compared with PMO alone. Cytotoxicity of the surfactants except for Mega10 was much lower than that by Endoporter in vitro and not clearly observed in vivo unde.. View more»

    DOI: 10.35248/2157-7439.20.11.553

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