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L Schoderboeck, S Riad, A M Bokor, H E Wicky, M Strauss, M Bostina, M J Oswald, R M Empson, and S.M. Hughes., Gene Therapy, 2015
Gene therapy is coming of age with the first gene therapy drug approved in Europe in 2012 and results of several other clinical trials recently released. Gene therapy (repairing or replacing defective genes) in the nervous system presents interesting challenges, especially when the gene therapy would cause major damage in any cells other than the specific target. In this study we modified a viral vector to deliver modified genes only to brainstem and cerebral cortex neurons after injection into the spinal cord. These new vectors are now being used to study the role of specific proteins in a group of cortical neurons involved in motor function. This work is part of a project funded by the Marsden Fund to Dr Hughes (Biochemistry) and A/Prof Empson (Physiology) with postdoctoral fellow Lucia Schoderböck and ARF Hollie Wicky (Hughes lab).