Special Session: Gene TherapyGene Therapy: When Desires Precede the Evidence By design gene therapy is predicated on personalized precision medicine where a given therapeutic strategy restores normal gene expression patterns, results in no insertional mutagenesis (no cancers), no innate/adaptive immune responses (no deaths), with no off-target effects (no star-activity) in a biologic milieu stagged with stereotypical endogenous DNA repair mechanisms that facilitates therapeutic correction of non-nested genes. However, today these predications are simply therapeutic desires that we have allowed to precede biologic evidence. Therefore, the current presentation will reveal the biologic foundations of gene therapy by contrasting current therapeutic desires against current biologic evidence. This is particularly important because, we can only unlock the true therapeutic potential of gene therapy when our therapeutic desires are circumscribed by biologic evidence.
Dr. O’neil W. Guthrie completed research training in molecular biology and audiology at the University of Pittsburgh where he earned a Ph.D. He then completed postdoctoral training in molecular genetics at Duke University where he was awarded the Hargett Cell Biology Fellowship. In addition to his research credentials, he is also a licensed clinical audiologist with decades of clinical experience. In addition to his basic science research program, Dr. Guthrie is also interested in translational research that improves clinical outcomes.
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