Glycogen Storage Disease (GSD) was almost universally fatal until 1971 when the first effective therapy was discovered, cornstarch. While this may not sound sophisticated, it turned this from a fatal disease to one where people are thriving. When treated properly, patients are healthy; when suboptimal treatment occurs, however, patients may develop permanent neurologic damage, seizures, or even die. Even with modern therapy, complications remain common including hepatic adenomas, liver cancer, renal failure, osteoporosis, anemia, and inflammatory blowel disease.
In 2005, Dr. Weinstein moved from Harvard Medical School and Children's Hospital Boston to the University of Florida in order to pursue a dream of performing gene therapy in glycogen storage disease. The lack of a vet school in Boston made it impossible to perform the studies, and the University of Florida offered outstanding research infrastructure. Since arriving 5 years ago, core teams have been created in 10 areas.
- Gene Therapy Team
- Adult Stem Cell Team
- Nerologic Disorders and Attention Deficit Hyperactivity Team
- Treatment Trial Team
- Cardiovascular Team
- Exercise Team
- Anemia Team
- Inflammatory Bowel Disease Team
- Liver Team
- Dental Research Team