Widespread suppression of huntingtin with convection-enhanced delivery of siRNA

@article{Stiles2012WidespreadSO,
  title={Widespread suppression of huntingtin with convection-enhanced delivery of siRNA},
  author={David K. Stiles and Zhiming Zhang and Pei Ge and Brian H. Nelson and Dinah W. Y. Sah},
  journal={Experimental Neurology},
  year={2012},
  volume={233},
  pages={463-471}
}
  • David K. Stiles, Zhiming Zhang, +2 authors Dinah W. Y. Sah
  • Published in Experimental Neurology 2012
  • Biology, Medicine
  • Huntington's disease is an autosomal dominant neurodegenerative disease caused by a toxic gain of function mutation in the huntingtin gene (Htt). Silencing of Htt with RNA interference using direct CNS delivery in rodent models of Huntington's disease has been shown to reduce pathology and promote neuronal recovery. A key translational step for this approach is extension to the larger non-human primate brain, achieving sufficient distribution of small interfering RNA targeting Htt (siHtt) and… CONTINUE READING

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