Author: Julia Evangelou Strait

Researchers at the School of Medicine have identified an unusual cause of the lysosomal storage disorder called mucolipidosis III, at least in a subset of patients. Unlike most genetic diseases that involve dysfunctional or missing proteins, the culprit is a normal protein that ends up in the wrong place.

Researchers at the School of Medicine have demonstrated a new approach to treating muscular dystrophy. Mice with a form of the disease showed improved strength and heart function when treated with nanoparticles loaded with rapamycin, an immunosuppressive drug recently found to improve recycling of cellular waste.

Researchers at the School of Medicine have shown that two major pools of immune cells are at work in the heart. Both belong to a class of cells known as macrophages. One appears to promote healing, while the other likely drives inflammation, which is detrimental to long-term heart function.

Andrew S. Yoo, PhD, a researcher at the School of Medicine, has received a Presidential Early Career Award for Scientists and Engineers, the highest honor given by the U.S. government to independent researchers early in their careers.

Working in mice, School of Medicine researchers report developing a gene delivery method long sought in the field of gene therapy: a deactivated virus carrying a gene of interest that can be injected into the bloodstream and make its way to the right cells. In this proof-of-concept study, they targeted tumor blood vessels in mice without affecting healthy tissues.

Bariatric surgery is effective at helping patients lose weight and improve obesity-related conditions such as diabetes, high blood pressure and sleep apnea, according to a review of published studies. Complications are possible following these procedures, but death rates generally are low, according to School of Medicine researchers who performed the analysis.

Patients who experience ‘chemobrain’ following treatment for breast cancer show disruptions in brain networks that are not present in patients who do not report cognitive difficulties, according to School of Medicine researchers.

Human breast tumors transplanted into mice are excellent models of metastatic cancer and are providing insights into how to attack breast cancers that no longer respond to the drugs used to treat them, according to research led by Matthew J. Ellis, MD, PhD, from Washington University School of Medicine in St. Louis.

Widely used treatments for type 2 diabetes have different effects on the hearts of men and women, even as the drugs control blood sugar equally well in both sexes, according to researchers at the School of Medicine. The investigators used PET scans to measure heart and whole-body metabolism in patients taking common diabetes drugs. Pictured are researchers Janet B. McGill, MD, and Robert J. Gropler, MD.

In search of better cancer treatments, Xiaowei Wang, PhD, and his colleagues at the School of Medicine have designed synthetic molecules that combine the advantages of two experimental RNA therapies.