About 79% of clinical trial participants experienced measurable improvement after receiving experimental, CRISPR-based gene editing that is designed to fix a rare form of blindness, according to a paper published today in the New England Journal of Medicine.

"This trial shows CRISPR gene editing has exciting potential to treat inherited retinal degeneration," said Mark Pennesi, M.D., Ph.D., a corresponding author on the paper, an ophthalmologist and Oregon Health & Science University's lead scientist for the Phase 1/2 BRILLIANCE trial.

Neuroblastoma is a common pediatric cancer that originates in developing nerve cells outside the brain. While increasingly intensive treatments have improved the survival of patients with high-risk neuroblastoma, currently more than 40% of patients do not survive.

New research from the University of Chicago shows the potential for a promising new approach to treating neuroblastoma by targeting RNA modifications associated with the disease.

Working with human breast and lung cells, Johns Hopkins Medicine scientists say they have charted a molecular pathway that can lure cells down a hazardous path of duplicating their genome too many times, a hallmark of cancer cells.

The findings, published May 3 in Science, reveal what goes wrong when a group of molecules and enzymes trigger and regulate what's known as the "cell cycle," the repetitive process of making new cells out of the cells' genetic material.

Many bacteria produce substances to gain an advantage over competitors in their highly competitive natural environment. Researchers at the University Hospital Bonn (UKB), the University of Bonn and the German Center for Infection Research (DZIF) have discovered a new so-called lantibiotic, namely epilancin A37. It is produced by staphylococci that colonize the skin and acts specifically against their main competitors there, the corynebacteria.

Researchers at UNC Lineberger Comprehensive Cancer Center, after decades of research efforts, have developed a mouse model of Kaposi sarcoma that could be key to the development of new drugs to treat the disease. Kaposi sarcoma is a cancer that is the most common cancer in people living with HIV.

The findings appeared in Cell Host & Microbe.

A healthy lifestyle may offset the effects of life-shortening genes by more than 60%, suggests an analysis of the findings from several large long term studies, published online in the journal BMJ Evidence Based Medicine.

While genes and lifestyle seem to have an additive effect on a person's lifespan, an unhealthy lifestyle is independently linked to a 78% heightened risk of dying before one’s time, regardless of genetic predisposition, the research indicates.

In a landmark study, an international consortium led by researchers at Children's Hospital of Philadelphia (CHOP) published the final results of a key clinical trial of the gene therapy CASGEVY (exagamglogene autotemcel) for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs). The study found that 96.7% of patients in the study did not have any vaso-occlusive crises (VOCs) - a blockage that results in lack of oxygen and painful episodes - for at least one year, and 100% were able to remain hospitalization-free for the same length of time.