Systemic delivery of antisense oligomer in animal models and its implications for treating DMD.

@article{Lu2012SystemicDO,
  title={Systemic delivery of antisense oligomer in animal models and its implications for treating DMD.},
  author={Qi Long Lu and Bo Wu},
  journal={Methods in molecular biology},
  year={2012},
  volume={867},
  pages={393-405}
}
Antisense oligomer (AO)-mediated splicing modulation for treating DMD demands a systemic administration of AOs as pharmacological drugs to achieve effective prevention of disease progression and to improve quality and longevity of patient life. Three routes, namely, intravenous injection (IV), intraperitoneal injection (IP), and subcutaneous injections (SC), of systemic delivery of AOs have been examined in animal models, and two of them, IV and SC, are being applied in the ongoing clinical… CONTINUE READING