Our research on childhood onset neurodegenerative diseases is focused on experiments to better understand the biochemistry and cell biology of proteins deficient in these disorders, and to develop small molecule or gene therapy based strategies for therapy. In recent work, we demonstrated that the application of recombinant viral vectors to various models of storage disease reversed CNS deficits and improved life span. We continue to develop novel vector systems to improve therapeutic outcomes.
The Camire Lab is interested in understanding the components of the blood coagulation system, how they interface with activated cells, and how disturbances in their function lead to bleeding and thrombosis. The lab is also interested in developing novel therapeutic approaches (protein, gene-based, small molecule) to mitigate these events which are major causes of morbidity and mortality worldwide.
The Ahrens-Nicklas lab combines translational studies in patients with molecular, biochemical, and electrophysiologic studies working with animal models to develop novel therapies for rare inherited pediatric disorders.
Dr. George's clinical and research interests are in the development of novel therapeutics for hemophilia. The George Laboratory studies the molecular basis of coagulation with specific emphasis on the intrinsic tenase enzyme complex.