Developing Treatments for Pediatric Brain Diseases through Genomics
Event Details
- Type
- Friday Lecture Series
- Speaker(s)
-
Joseph Gleeson, M.D., professor of neurosciences and pediatrics, University of California, San Diego; investigator, Howard Hughes Medical Institute
- Speaker bio(s)
-
Dr. Gleeson's lab is focused on identifying new causes and treatments for neurodevelopmental disabilities in children. Both diagnosis and treatment for children suffering from these disabilities have lagged behind other fields of medicine for many years, in part due to the inaccessibility of diseased cells and the fact that by the time an affected child is born the brain is mostly developed.
By combining brain imaging, biomarkers and genomics, it is possible to determine specific diagnoses in many cases. By employing induced pluripotent stem cell technology in cells from patients with genetically proven disease, it is possible to fundamentally change the way we understand mechanisms and develop treatments.
The advent of genomics is beginning to change the landscape of treatment opportunities for these diseases. Careful selection of undiagnosed cases for genomic evaluation has revealed the power of unbiased “exome” sequencing to improve diagnosis and alter patient management. From the Gleeson lab's cohort of over 2,000 families with likely genetic forms of neurodevelopmental disabilities we have uncovered a host of new genetic causes, some predicted to be treatable. In this lecture, Dr. Gleeson will review recent progress in exome sequencing and highlight potentially treatable diseases, an exciting possibility for some children with these disabilities, once thought to be undiagnosable and untreatable.
Dr. Gleeson received his M.D. from the University of Chicago Pritzker School of Medicine in 1991 and completed a pediatrics residency and a neurology fellowship at Children’s Hospital in Boston. He did a research fellowship from 1995 to 1999 at Harvard Medical School. In 1999 he joined the faculty of the University of California, San Diego, where he is now full professor and head of the Neurogenetics Laboratory. Among several honors, Dr. Gleeson was awarded the Klingenstein Award in the Neurosciences in 2001 and the Burroughs Wellcome Fund Award in Translational Research in 2005. - Open to
- Public
- Reception
- Refreshments, 3:15 p.m. - 3:45 p.m., Abby Lounge
- Contact
- Gloria Phipps
- Phone
- (212) 327-8967
- Sponsor
-
Gloria Phipps
(212) 327-8967
phippsg@rockefeller.edu