Leslie Thompson, Ph.D.
Professor
Biography
Have been committed to Huntington’s Disease Research for over 20 years, beginning with the collaborative search for the disease gene. Also a participant in the Venezuela Huntington’s Disease research group. Dr. Thompson is an alumni of UCI as a graduate student and postdoctoral fellow, before becoming a faculty member.
Research in Lay Terms
Huntington’s disease (HD) is a devastating degenerative brain disease that inevitably leads to death. HD has no effective treatment or cure and symptoms unstoppably progress for 15-20 years, with onset typically striking in midlife. Because HD is genetically dominant, the disease has a 50% chance of being inherited by the children of patients. Because HD is genetic and the mutation known, a diagnosis can be made with certainty and clinical applications of hESCs and iPSCs may provide insights into treating this and other brain diseases that are not caused by a single, known mutation. Trials in mice with human stem cells are in progress. Banks of stem cells derived from patient skin samples are being developed and studied for HD symptoms in a dish for studies of the disease and drug screens.
Research Focus
The laboratory research focuses on Huntington’s disease, a devastating neurodegenerative disease and on FGFR3-associated forms of cancers. The lab is using stem cell approaches to investigate these disorders and identify potential therapeutic targets or treatments.
Research Details
Since the discovery of the HD gene, Dr. Thompson’s work has focused on understanding pathogenic mechanisms in HD with the goal of slowing or stopping progression of disease.Her laboratory has made significant progress toward understanding the role of the mutant Huntingtin protein in the disease process and has developed therapeutic strategies that may aid in treating HD. This work includes investigations of transcriptional dysregulation, signal transduction pathways altered in HD, altered degradation of the disease protein and modifications of the mutant protein that impact disease. Recent efforts have focused on the use of human embryonic stem cells and iPS cells to more closely replicate disease phenotypes to better understand the underlying mechanisms and for use in drug development, as well as for transplantation purposes. Similar approaches are being used to provide novel therapeutic targets in FGFR3-related cancers and again human stem cells are providing new and exciting opportunities to study the disease.
