The CRISPR genome editing technique has been used to activate a close relative of the gene that is mutated in muscular dystrophy type 1A, preventing development of the disease in mouse-model neonates and reversing symptoms in mice with established pathology.

MEXICO CITY – Ten years after the RV144 "Thai trial" was the first to show that an effective HIV vaccine was possible, three efficacy trials for HIV vaccines are once again underway.

Anorexia nervosa, Cynthia Bulik told BioWorld, has a long-term recovery rate of 25% and “the highest fatality rate of any psychiatric illness.” Those dismal statistics hint at the challenging nature of anorexia nervosa, but also at the shortcomings of current treatment options.

Scientists from the Wellcome Trust Sanger Institute have shown that antioxidant treatment could reverse the competitive advantage of p53-mutated cells in the esophagus of transgenic mice. 

Researchers from Johns Hopkins University School of Medicine have developed a machine learning program that could score the risk of pancreatic cysts and recommend one of three treatment strategies – surgery, watchful waiting or discharge without follow-up – more accurately than current methods. The program could potentially reduce the number of unnecessary surgeries performed on pancreatic cysts with little to no potential of turning cancerous.

A team at Dartmouth University has shown that in fruit flies, stressful experiences could lead to epigenetic changes that led to a preference for ethanol-rich foods for several generation of offspring. 

To hear Peter Sorger tell it, the reproducibility crisis is a good news/bad news situation.His team's "strangely simple and encouraging message" about the reproducibility crisis, Sorger told BioWorld, is that "we know exactly how to solve it... In the totally ordinary doing of science, you can figure out how to make an assay reproducible." Sources of variability "can be subjected to empirical analysis, and you can develop reproducible protocols, with some effort." The bad news is that what bedevils reproducibility is harder to fix than quality control. "The current incentive structure of the scientific enterprise," Sorger said, "is not designed to encourage reproducible science."

Despite the arrival of FDA-approved tissue-agnostic targeted cancer therapies, there is increasing recognition that the response of tumors that are driven by the same oncogene differs according to their location.

Despite its rather specific name, macrophage migration inhibitory factor (MIF) has multiple roles. One of those roles is to serve as a chaperone protein that helps the proper folding of superoxide dismutase (SOD1). 

In what may be the smallest double-blind, placebo-controlled clinical trials on record, researchers have shown that treating two individuals with drugs aimed at raising brain levels of glycine improved their psychotic symptoms.