Nucleic-acid therapeutics: basic principles and recent applications

@article{Opalinska2002NucleicacidTB,
  title={Nucleic-acid therapeutics: basic principles and recent applications},
  author={Joanna B. Opalinska and Alan M. Gewirtz},
  journal={Nature Reviews Drug Discovery},
  year={2002},
  volume={1},
  pages={503-514}
}
The sequencing of the human genome and the elucidation of many molecular pathways that are important in disease have provided unprecedented opportunities for the development of new therapeutics. The types of molecule in development are increasingly varied, and include antisense oligonucleotides and ribozymes. Antisense technology and catalytic nucleic-acid enzymes are important tools for blocking the expression of abnormal genes. One FDA-approved antisense drug is already in the clinic for the… 
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