Gene Therapy Research Unit

A joint initiative of The Children's Hospital at Westmead and Children's Medical Research Institute

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Gene therapy for diseases of nerve and muscle J. Fleming, A. Spinoulas, E. Kizana, S. Ginn and I. Alexander Recent advances in gene transfer technology have placed a limited but exciting number of therapeutic possibilities within reach of existing vectors. Prominent among these possibilities are diseases and pathophysiological states for which the genetic and phenotypic modification of relatively modest cell numbers, at defined anatomical locations, has therapeutic potential. Guided by this philosophy we have identified two pathophysiological processes that may be amenable to molecular based therapies using contemporary vectors. The dorsal root ganglia (DRG) encapsulate the cell bodies of the peripheral sensory neurons that mediate normal and abnormal sensory signals. The DRG are affected by a number of inherited and acquired conditions and by virtue of their discrete anatomical location, lend themselves to focal therapies such as gene therapy. We are interested in modifying the pathophysiology of Friedreich's ataxia and other peripheral neuropathies at the level of the DRG and also in modulating noxious sensation associated with various neuropathic pain syndromes. The second area of research interest is based on the focal genetic modification of the cardiac conducting system and myocardium in order to restore conduction in regions of conduction block and diminish or ablate conduction in key regions involved in the initiation and maintenance of cardiac arrhythmias.

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