Guanine analogues enhance antisense oligonucleotide-induced exon skipping in dystrophin gene in vitro and in vivo.

@article{Hu2010GuanineAE,
  title={Guanine analogues enhance antisense oligonucleotide-induced exon skipping in dystrophin gene in vitro and in vivo.},
  author={Yihong Hu and Bo Wu and Allen Zillmer and Peijuan Lu and Ehsan Benrashid and Mingxing Wang and Timothy Doran and Mona Shaban and Xiaohua Wu and Qi Long Lu},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2010},
  volume={18 4},
  pages={
          812-8
        }
}
Exon skipping has demonstrated great potential for treating Duchenne muscular dystrophy (DMD) and other diseases. We have developed a drug-screening system using C2C12 myoblasts expressing a reporter green fluorescent phosphate (GFP), with its reading frame disrupted by the insertion of a targeted dystrophin exon. A library of 2,000 compounds (Spectrum collection; Microsource Discovery System) was screened to identify drugs capable of skipping targeted dystrophin exons or enhancing the exon… CONTINUE READING

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Tween 85-Modified Low Molecular Weight PEI Enhances Exon-Skipping of Antisense Morpholino Oligomer In Vitro and in mdx Mice

Molecular therapy. Nucleic acids • 2017
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