Conformational Diseases and Therapeutics Research Laboratory
The Conformational Diseases and Therapeutics Research (CDTR) Lab works on understanding the mechanisms of protein folding and misfolding, and to decipher how these processes are related to normal physiology and disease using chemical, biophysical and cell biology approaches. The lab is currently working on the following protein folding disorders:
Huntington's disease, an incurable and degenerative disease of the nervous system for which no cure exists. It is progressive and causes uncontrolled movements, emotional problems, and loss of thinking ability.
Alpha 1 antitrypsin deficiency, a condition that leads to emphysema and liver diseases as the responsible protein forms polymers in the hepatocytes (liver cells) preventing it to fulfill its normal biological function: protecting the lungs.
Image of Diastase-resistant, PAS-positive polymers of mutant alpha1-antitrypsin (pink dots) in liver cells.
The CDTR lab works also on identifying and searching for small molecules that can lead to potential therapeutics via high-throughput screening assays. To be able to deliver these potent drugs, a Biofermentation Nanoparticle Platform has been set up.
Principal Techniques Used
Principal Investigator
Valerie Berthelier, PhD
Assistant Professor, Department of Medicine
Director, Conformational Diseases and Therapeutics
The Current Research Group
Kasey Estenson (PhD student)
Jessica Voiles (Undergraduate)
Sarah Lucas (Senior High-School)
The Collaborators
ORNL
Jennifer Morell-Falvey (BioSciences Division)
Hugh O’Neill (Biology and Soft Matter Division)
Christopher Stanley (Neutron Sciences)
The University of Tennessee
Jerome Baudry (BCMB)
UT Institute of Biomedical Engineering
Christopher Stephens, PhD