Pharmacology of a central nervous system delivered 2'-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates.

@article{Rigo2014PharmacologyOA,
  title={Pharmacology of a central nervous system delivered 2'-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates.},
  author={Frank W Rigo and Seung Jong Chun and Daniel A. Norris and Gene H Hung and Sam Pin Lee and John Matson and Robert A Fey and Hans J. Gaus and Yimin Hua and John S. Grundy and Adrian R. Krainer and Scott P. Henry and C. F. Bennett},
  journal={The Journal of pharmacology and experimental therapeutics},
  year={2014},
  volume={350 1},
  pages={46-55}
}
Spinal muscular atrophy (SMA) is a debilitating neuromuscular disease caused by the loss of survival of motor neuron (SMN) protein. Previously, we demonstrated that ISIS 396443, an antisense oligonucleotide (ASO) targeted to the SMN2 pre-mRNA, is a potent inducer of SMN2 exon 7 inclusion and SMN protein expression, and improves function and survival of mild and severe SMA mouse models. Here, we demonstrate that ISIS 396443 is the most potent ASO in central nervous system (CNS) tissues of adult… CONTINUE READING

Citations

Publications citing this paper.
Showing 1-10 of 50 extracted citations

References

Publications referenced by this paper.
Showing 1-10 of 53 references

Similar Papers

Loading similar papers…