A new gene therapy appears to serve as a functional cure for the most common type of hemophilia, early clinical trial results indicate.
Patients who received the one-time intravenous therapy continue to have a more than 90% decrease in bleeding events two to three years after their initial treatment, researchers reported Jan. 1 in the New England Journal of Medicine.
Last year, a scientist from China created a storm of controversy when he claimed he'd used gene-editing technology to create "designer" twin babies. Now, a new study is highlighting one of the dangers of that endeavor.
Researchers have found that the gene mutation the scientist used -- affecting a gene called CCR5 -- is associated with a shorter life span: People who carry two copies ...
The controversy over a Chinese scientist who claimed he created gene-edited babies has prompted the U.S. National Institutes of Health to join an international moratorium on such research.
"Today, leading scientists and ethicists from seven countries have called for an international moratorium on the use of genetic editing to modify the human germline for clinical purposes," NIH Direc...
The supply of donor organs for infants needing a heart transplant is critically low, but researchers have taken a first step toward using pig hearts to fill the need.
The concept of using animal organs to save human lives has been around for years. With donor organs in short supply, the hope is that animal organs can keep patients alive while they await a human donor.
An experimental gene therapy for Parkinson's disease seems to work by rewiring key areas of the brain, a new study finds.
The researchers focused on 15 Parkinson's patients who, in an earlier trial, had received so-called GAD gene therapy. GAD is an enzyme that spurs the production of a brain chemical involved in movement control.
A new gene therapy might help improve motor symptoms in people with Parkinson's disease who aren't responding to other therapies, an early study has found.
"This is not a cure of Parkinson's disease," said James Beck, chief scientific officer of the Parkinson's Foundation. "This is a potentially good treatment for symptom control. It provides an additional way of providing dopamine t...