Antisense effects in the cell nucleus: modification of splicing

Curr Opin Mol Ther. 2001 Jun;3(3):229-34.


The surprisingly small number of human genes, which has recently been estimated to be approximately 30,000, suggests that RNA processing, and in particular alternative RNA splicing, is in large part responsible for the diversity of gene products in human and mammalian cells. The ability to manipulate alternative splicing using antisense oligonucleotides, as demonstrated in several studies during the past year, makes it an important and attractive approach to altering gene expression. A review of these studies leads to the conclusion that antisense oligonucleotides, whether designed to affect the cytoplasmic mRNA or nuclear pre-mRNA, function predominantly in the nucleus and not the cytoplasm.

Publication types

  • Review

MeSH terms

  • Alternative Splicing*
  • Animals
  • Cell Nucleus / genetics
  • Cell Nucleus / metabolism*
  • Gene Expression Regulation*
  • Genetic Diseases, Inborn / drug therapy
  • Humans
  • Oligonucleotides, Antisense / genetics
  • Oligonucleotides, Antisense / metabolism*
  • Oligonucleotides, Antisense / therapeutic use
  • Thionucleotides / genetics
  • Thionucleotides / metabolism*
  • Thionucleotides / therapeutic use


  • Oligonucleotides, Antisense
  • Thionucleotides