Dr. Rubenstein's research focuses on basic and translational studies of novel means to improve outcomes in cystic fibrosis. He initially focused on correcting the dysfunction of mutant cystic fibrosis transmembrane conductance regulator (CFTR) proteins, which led him to study how molecular chaperones regulate the biogenesis and trafficking of CFTR and other proteins that are relevant to cystic fibrosis.
Dr. Marks investigates the molecular mechanisms underlying the formation of cell type-specific lysosome-related organelles; the assembly, delivery and function of their contents; and how these processes are impacted by genetic diseases.
Researchers at The Children’s Hospital of Philadelphia reported their latest results from their studies of an investigational personalized cell therapy for a highly aggressive form of cancer, acute lymphoblastic leukemia (ALL).
By being named a 2013 Pew Scholar, the Children's Hospital of Philadelphia's Claudio Giraudo, PhD, joins a "prestigious community of Nobel laureates, MacArthur fellows, Albert Lasker Basic Medical Research Award winners, and hundred of other pioneers who earned Pew grants at the start of their careers." The Pew Scholars are each awarded $240,000 over four years to conduct their research,
The Marks Lab aims to dissect the molecular mechanisms underlying the formation of cell type-specific lysosome-related organelles and the assembly, delivery, and function of their contents, and to understand how these processes are impacted by genetic diseases.