Phase I Clinical Trial of Systemically Administered TUSC2(FUS1)-Nanoparticles Mediating Functional Gene Transfer in Humans

  title={Phase I Clinical Trial of Systemically Administered TUSC2(FUS1)-Nanoparticles Mediating Functional Gene Transfer in Humans},
  author={Charles Lu and David J. Stewart and James S. J. Lee and Lin Qin Ji and Rajagopal Ramesh and Gitanjali Jayachandran and Mar{\'i}a Isabel N{\'u}{\~n}ez and Ignacio Wistuba and Jeremy J. Erasmus and Marshall E. Hicks and Elizabeth Ann Grimm and James M. Reuben and Veerabhadran Baladandayuthapani and Nancy Templeton and John D. McMannis and J. L. A. Roth},
  booktitle={PloS one},
BACKGROUND Tumor suppressor gene TUSC2/FUS1 (TUSC2) is frequently inactivated early in lung cancer development. TUSC2 mediates apoptosis in cancer cells but not normal cells by upregulation of the intrinsic apoptotic pathway. No drug strategies currently exist targeting loss-of-function genetic abnormalities. We report the first in-human systemic gene therapy clinical trial of tumor suppressor gene TUSC2. METHODS Patients with recurrent and/or metastatic lung cancer previously treated with… CONTINUE READING


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