Center for 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.
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Weihang (Valerie) Chai, PhD
Director, Center for Genetic Diseases
- Weihang (Valerie) Chai, Ph.D.
- Molecular Basis of Coats Plus disease
- Molecular mechanisms for maintaining genome stability under replication stress
- Understanding the genetic disposition of CST deficiency in colorectal diseases and carcinogenesis using mouse models
- Mechanisms for PARP inhibitor resistance in BRCA2-deficient cells
- David M. Mueller, Ph.D.
- 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.
- JongSun Lee, Ph.D.
- Studies the role of long non-coding RNAs in gene regulation, ribosome biogenesis, and translational control
- Examines how dysregulated RNA pathways contribute to cancer and other human diseases, with a particular focus on kidney and gastrointestinal cancers
- Combines functional genomics, molecular biology, and RNA biochemistry to define disease mechanisms
- Seeks to identify new RNA-based therapeutic and diagnostic opportunities
- Paige Arneson-Wissink, Ph.D.
- Epigenetic regulation of metabolic gene expression
- Metabolic deficits associated with pancreatic cancer cachexia
- Persistent deficits in metabolism and body composition after cancer recovery
- Mouse models of pancreatic cancer cachexia
- Jessica Centa, Ph.D.
- Develops therapeutic antisense oligonucleotides (ASOs) that modulate RNA splicing for genetic diseases such as CLN3 Batten disease and cystic fibrosis
- Designs personalized nucleic acid-based therapies for rare genetic disorders
- Utilizes electrophysiological assays to measure ion channel activity (such as CFTR), validating treatment efficacy in disease-affected cells, including cystic fibrosis
- Fang Wang, Ph.D.
- Study how STN1 loss shapes immune responses in the colon.
- Elucidate the dynamic, context-dependent roles of B cells in hepatocellular carcinoma progression.
- Robert J. Bridges (Professor Emeritus)
- 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.