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Tailoring mTOR-based therapy: molecular evidence and clinical challenges

    Gaetano Santulli

    Department of Physiology & Cellular Biophysics, The Clyde & Helen Wu Center for Molecular Cardiology, Columbia University Medical Center, New York, NY 10032, USA

    &
    Hana Totary-Jain

    * Author for correspondence

    Department of Physiology & Cellular Biophysics, The Clyde & Helen Wu Center for Molecular Cardiology, Columbia University Medical Center, New York, NY 10032, USA.

    Published Online:https://doi.org/10.2217/pgs.13.143

    The mTOR signaling pathway integrates inputs from a variety of upstream stimuli to regulate diverse cellular processes including proliferation, growth, survival, motility, autophagy, protein synthesis and metabolism. The mTOR pathway is dysregulated in a number of human pathologies including cancer, diabetes, obesity, autoimmune disorders, neurological disease and aging. Ongoing clinical trials testing mTOR-targeted treatments number in the hundreds and underscore its therapeutic potential. To date mTOR inhibitors are clinically approved to prevent organ rejection, to inhibit restenosis after angioplasty, and to treat several advanced cancers. In this review we discuss the continuously evolving field of mTOR pharmacogenomics, as well as highlight the emerging efforts in identifying diagnostic and prognostic markers, including miRNAs, in order to assess successful therapeutic responses.

    Papers of special note have been highlighted as: ▪ of interest ▪▪ of considerable interest

    References

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