Genetic Diseases
We investigate genetic diseases and work to understand the mechanisms of dysfunction and to discover ways to treat disease using small molecule and nucleic acid-based approaches.
In this section
In this section
Michelle L. Hastings, PhD
Director, Center for Genetic Diseases
- Michelle L. Hastings – Center Director
- Mechanisms of molecular dysfunction that contribute to our understanding of disease and to aid in the identification of targets for therapeutic interventions.
- Antisense oligonucleotide technology and small molecule compounds for the treatment of genetic diseases, including Batten disease, cystic fibrosis, Usher syndrome, Alzheimer's and Parkinson’s disease.
- Robert J. Bridges
- Identification of potential drug candidates for the treatment of cystic fibrosis.
- Probing human nasal epithelial cells for in vitropharmacogenomic studies for the treatment of cystic fibrosis.
- Neil Bradbury
- The regulation of membrane protein trafficking in cystic fibrosis.
- Probing human nasal epithelial cells for in vitropharmacogenomic studies for the treatment of cystic fibrosis.
- David M. Mueller
- The structure and functional mechanism of the mitochondrial F1FO-ATPase. The structural basis for drugs and inhibitors that target the ATP synthase.
- The structure and function of Cln3 - the gene defective in the juvenile form of Batten disease, a neurodegenerative disease of the brain.