Silencing disease genes in the laboratory and the clinic.

@article{Watts2012SilencingDG,
  title={Silencing disease genes in the laboratory and the clinic.},
  author={Jonathan K. Watts and David R. Corey},
  journal={The Journal of pathology},
  year={2012},
  volume={226 2},
  pages={
          365-79
        }
}
Synthetic nucleic acids are commonly used laboratory tools for modulating gene expression and have the potential to be widely used in the clinic. Progress towards nucleic acid drugs, however, has been slow and many challenges remain to be overcome before their full impact on patient care can be understood. Antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs) are the two most widely used strategies for silencing gene expression. We first describe these two approaches and… CONTINUE READING
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