Vanderbilt University Medical Center investigators are reporting success with a novel gene therapy approach. Working with cells grown in the laboratory, the group is the first to repair a defective gene and demonstrate that the resulting protein product is functional, said Dr. Alfred L. George Jr., senior author of a study published Dec. 15 in the Journal of Clinical Investigation.

Although use of the approach in patients is still years in the future, the findings are an important step in sh

Scientists at the University of Virginia School of Medicine have discovered a new contributor to atherosclerosis, the most common form of hardening of the arteries. Marked by cholesterol-calcium-lipid deposits, atherosclerosis is the main cause of heart attacks, the number one killer in the U.S. Doctors at U.Va. say research on mice has determined for the first time that activated platelets circulating in the blood, long understood as markers for atherosclerosis, really serve as participants in the p

Researchers at Cedars-Sinai Medical Center’s Maxine Dunitz Neurosurgical Institute in Los Angeles have combined a special protein that targets cancer cells with neural stem cells (NSC) to track and attack malignant brain tumor cells. Results of their study appear in the Dec. 15 issue of Cancer Research.

Glioblastoma multiforme, or gliomas, are a particularly deadly type of brain tumor. They are highly invasive with poorly defined borders that intermingle with healthy brain tissue, maki

A collaboration of scientists from Harvard Medical School and Dartmouth Medical School has developed a new mouse model of lipoatrophic diabetes, and highlighted leptin therapy as a successful tool to combat this rare form of type II diabetes.

Lipoatrophic diabetes mellitus is characterized by a lack of subcutaneous fat (lipoatrophy), high blood sugar (hyperglycemia), and high blood insulin (hyperinsulinemia). Because patients with lipoatrophic diabetes are insulin-resistant, although high l

Dr. Mario Capecchi and colleagues at the University of Utah and the Department of Veterans Affairs Medical Center (Salt Lake City, UT) have discovered that a gene called xanthine oxidoreductase, or XOR for short, is required for lactation in female mice. This previously unidentified role for XOR in lactation reveals a possible genetic basis for the lactation difficulties experienced by nearly 5% of women.

XOR was originally identified as encoding an enzyme involved in purine catabolism (the

A key role in synchronizing daily rhythms to the day/night cycle has been traced to a light-sensitive protein in the eye, by knocking out the gene that codes for it. Mice lacking a gene for the photopigment melanopsin show a dramatic deficiency in their ability to regulate their circadian rhythms by light. The discovery, by National Institute of Mental Health (NIMH) grantees, helps unravel the heretofore elusive mechanisms by which day/night cycles regulate such rhythms in mammals. NIMH grantees Igna