The broad focus of our research is the development of promising innovative approaches to treat neurological disorders. By studying the mechanisms of diseases in different animal models we are better able to design rational drugs and biologics that target upstream events in pathophysiology. We are able to test novel vector designs with in vivo models as part of the process of bringing new advancements in virology and molecular biology to the clinical setting. In a close partnership with the UNC Gene Therapy Center we are optimizing recombinant Adeno-associated viral vectors for use in clinical gene therapy studies.
In addition to clinical research on Gene Therapy of Canavan Disease, the CGTC is engaged in basic research into the mechanistic basis of neurological disease. The laboratory is currently actively investigating models of Canavan Disease and other neurodegenerative diseases and is pursuing potential treatments for these diseases via the application of gene transfer, stem cell, and pharmacological technologies. Investigators at the CGTC have a long-standing interest in viral vectors as gene transfer vehicles and have access to techniques enabling the transfer of potentially any gene of interest into available disease models. Other research interests related to the study of diseases of the brain include the analysis of signal transduction mechanisms involved in the regulation of fundamental cellular processes such as programmed cell death and regulation of the cell cycle, and investigation into the molecular basis of cognition.