Researchers in the Pennsylvania Muscle Institute (PMI), based at the Perelman School of Medicine at the University of Pennsylvania have received $9 million over the next five years from the National Institute of General Medical Sciences to investigate the biology of cellular motors.

Using a zebrafish model of a human genetic disease called neurofibromatosis (NF1), a team from the Perelman School of Medicine at the University of Pennsylvania has found that the learning and memory components of the disorder are distinct features that will likely need different treatment approaches.

Hoping to encourage sufficient investments by pharmaceutical companies in expensive gene therapies, which often consist of a single treatment, a Penn researcher and the chief medical officer of CVS Health outline an alternative payment model in this month’s issue of Nature Biotechnology.

Over the years researchers have made tremendous strides in the understanding and treatment of cancer by searching genomes for links between genetic alterations and disease.

Treating the rare disease MPS I is a challenge. MPS I, caused by the deficiency of a key enzyme called IDUA, eventually leads to the abnormal accumulation of certain molecules and cell death. 

A Penn Medicine-developed drug has received orphan status in Europe this week for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare, life-threatening disease that causes anemia due to destruction of red blood cells and thrombosis.

Erika Holzbaur, PhD, a professor of Physiology in the Perelman School of Medicine at the University of Pennsylvania, has received the Javits Neuroscience Investigator Award from the National Institutes of Health.

Researchers at the Perelman School of Medicine at the University of Pennsylvania will receive $7.3 million over the next five years from the National Cancer Institute (NCI) to find new ways to treat esophageal cancer.

Actin is the most abundant protein in the body, and when you look more closely at its fundamental role in life, it’s easy to see why. It is the basis of most movement in the body, and all cells and components within them have the capacity to move: muscle contracting, heart beating, blood clotting, and nerve cells communicating, among many other functions.

In an analysis of small molecules called metabolites used by the body to make fuel in normal and cancerous cells in human kidney tissue, a research team from the Perelman School of Medicine at the University of Pennsylvania identified an enzyme key to applying the brakes on tumor growth.