Preclinical Justification of pbi-shRNA EWS/FLI1 Lipoplex (LPX) Treatment for Ewing's Sarcoma

  title={Preclinical Justification of pbi-shRNA EWS/FLI1 Lipoplex (LPX) Treatment for Ewing's Sarcoma},
  author={Donald D. Rao and Christopher M Jay and Zhaohui Wang and Xiuquan Luo and Padmasini Kumar and Hilary Eysenbach and Maurizio L. Ghisoli and Neil N. Senzer and John Nemunaitis},
  booktitle={Molecular therapy : the journal of the American Society of Gene Therapy},
The EWS/FLI1 fusion gene is well characterized as a driver of Ewing's sarcoma. Bi-shRNA EWS/FLI1 is a functional plasmid DNA construct that transcribes both siRNA and miRNA-like effectors each of which targets the identical type 1 translocation junction region of the EWS/FLI1 transcribed mRNA sequence. Previous preclinical and clinical studies confirm the safety of this RNA interference platform technology and consistently demonstrate designated mRNA and protein target knockdown at greater than… CONTINUE READING
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