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Our group is almost entirely focused on translational neuroscience, with major programs in brain cancer and Parkinson’s disease (PD). The long-term goal of our research team is to build powerful and practical solutions to the problems of delivery of therapeutics to the brain, whether they be gene therapy vectors, pharmaceuticals, cells, or recombinant proteins. All of our efforts are devoted toward the development of a unified technology that can accommodate many very different kinds of therapeutic agent into a single, easily controlled delivery system. To that end, we have developed an advanced type of delivery cannula that is biocompatible and resists reflux. We have also introduced the use of liposomes that can be visualized in real time by MRI, a technology that permits us to follow the progress of infusions of co-administered therapeutic agents. Careful integration of these different component technologies has provided us with a core delivery technology that permits us to delivery new kinds of therapies to many different areas of the brain with unprecedented control.
In addition to this technology, we have in place a team of experts in rodent and primate models of Parkinson’s disease adept at biochemical, histological, and behavioral studies. We have participated in the initiation of a clinical trial of a gene therapy for PD at UCSF and this experience has established strong pre-clinical development expertise within our group. We anticipate, therefore, the filing of further Investigational New Drug applications over the next 5 years.
Selected Publications
Bankiewicz, K. S., Forsayeth, J., Eberling, J. L., Sanchez-Pernaute, R., Pivirotto, P., Bringas, J., Herscovitch, P., Carson, R. E., Eckelman, W., Reutter, B. and Cunningham, J. (2006). Long-Term Clinical Improvement in MPTP-Lesioned Primates after Gene Therapy with AAV-hAADC. Mol Ther.
Forsayeth, J. R., Eberling, J. L., Sanftner, L. M., Zhen, Z., Pivirotto, P., Bringas, J., Cunningham, J. and Bankiewicz, K. S. (2006). A dose-ranging study of AAV-hAADC therapy in parkinsonian monkeys. Mol Ther 14: 571-7.
Saito, R., Krauze, M. T., Bringas, J. R., Noble, C., McKnight, T. R., Jackson, P., Wendland, M. F., Mamot, C., Drummond, D. C., Kirpotin, D. B., Hong, K., Berger, M. S., Park, J. W. and Bankiewicz, K. S. (2005). Gadolinium-loaded liposomes allow for real-time magnetic resonance imaging of convection-enhanced delivery in the primate brain. Exp Neurol.
Information last updated April 2007
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