Design of 2'-O-Me RNA/ENA chimera oligonucleotides to induce exon skipping in dystrophin pre-mRNA

Nucleic Acids Symp Ser (Oxf). 2004:(48):297-8. doi: 10.1093/nass/48.1.297.

Abstract

2'-O-Me RNA/ENA chimera oligonucleotides complementary to exon 45 and 46 of the dystrophin gene induced exon 45 and 46 skipping of the dystrophin pre-mRNA, respectively. The induction of exon skipping by the most effective 2'-O-Me RNA/ENA chimeras led to the expression of dystrophin in dystrophin-deficient myocytes by correcting the translational reading frames. Also, in the process of 2'-O-Me RNA/ENA chimera optimization to induce exon skipping in several exons, it was found that the optimized target sequences of the chimeras included guanosine- or adenosine-rich sequences that might function as spiking enhancer sequences (SES).

MeSH terms

  • Adenosine
  • Base Sequence
  • Drug Design
  • Dystrophin / genetics*
  • Dystrophin / metabolism
  • Exons / genetics*
  • Gene Expression Regulation
  • Guanosine
  • Humans
  • Nucleic Acid Heteroduplexes / chemistry
  • Nucleic Acid Heteroduplexes / metabolism*
  • Nucleic Acids / chemistry
  • Nucleic Acids / metabolism*
  • Oligonucleotides / chemistry
  • Oligonucleotides / metabolism*
  • RNA / chemistry
  • RNA / metabolism*
  • RNA Precursors / genetics*

Substances

  • Dystrophin
  • Nucleic Acid Heteroduplexes
  • Nucleic Acids
  • Oligonucleotides
  • RNA Precursors
  • Guanosine
  • RNA
  • Adenosine