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Ronald Rubenstein, MD, PhD

Attending Pulmonologist

Director, Cystic Fibrosis Center


Protein trafficking in cystic fibrosis. Dr. Rubenstein is pediatric pulmonologist interested in novel drug therapies for cystic fibrosis (CF). By understanding the molecular defects in the mutant CF conductance regulatory protein (CFTR), which is absent in the disease, Dr. Rubenstein and his colleagues aim to use novel pharmaceuticals to overcome these molecular defects and "repair" the dysfunctional CFTR. The researchers anticipate that such "protein repair" agents will improve the health of patients with CF.

Dr. Rubenstein and his colleagues have focused on the repair of the most common CFTR mutation, deltaF508-CFTR, which is not found at its appropriate location within epithelial cells. The researchers are studying one particular "protein repair" agent, sodium 4-phenylbutyrate (4PBA), which allows deltaF508-CFTR to reach the appropriate location in epithelial cells. They are using standard cell and molecular biology in vitro techniques in cultured cells and the Xenopus oocyte model expression system to assess the influence of 4PBA on proteins important in the intracellural trafficking of deltaF508-CFTR. As they identify 4PBA-regulated proteins that interact with deltaF508-CFTR, we aim to perform specific modulations of protein expression that will afford insight to the mechanism and repair of deltaF508-CFTR's aberrant intracellular trafficking. The researchers ultimately aim to extend and translate these observations into clinical trials of 4PBA and other "protein repair" agents.


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