Intracellular delivery of an anionic antisense oligonucleotide via receptor-mediated endocytosis

  title={Intracellular delivery of an anionic antisense oligonucleotide via receptor-mediated endocytosis},
  author={M d Rowshon Alam and Vidula R. Dixit and Hyunmin Kang and Zi-bo Li and Xiaoyuan Chen and JoAnn Trejo and Michael J Fisher and Rudolph L Juliano},
  booktitle={Nucleic acids research},
We describe the synthesis and characterization of a 5' conjugate between a 2'-O-Me phosphorothioate antisense oligonucleotide and a bivalent RGD (arginine-glycine-aspartic acid) peptide that is a high-affinity ligand for the alphavbeta3 integrin. We used alphavbeta3-positive melanoma cells transfected with a reporter comprised of the firefly luciferase gene interrupted by an abnormally spliced intron. Intranuclear delivery of a specific antisense oligonucleotide (termed 623) corrects splicing… CONTINUE READING


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