Gene therapy might soon offer a new option for children with a rare genetic disorder that damages tissues throughout the body, researchers are reporting.
In a study of eight children with the condition, called Hurler syndrome, researchers found that the gene therapy was safe over two years. It also showed potential for beating the current standard treatment, stem cell transplantation.
An experimental gene therapy to boost the effectiveness of the Parkinson's drug levodopa yielded promising results in mice, researchers report.
As the loss of dopamine-releasing neurons advances in late-stage Parkinson's, levodopa is less able to ease movement problems caused by the disease, which is a progressive disorder of the nervous system.
Nine of 10 patients with so-called "bubble boy" immune disease who received gene therapy about a decade ago are still disease-free, researchers report.
The gene therapy was developed at the University of California, Los Angeles (UCLA), to treat the rare and deadly immune system disorder formally known as adenosine deaminase--deficient severe combined immunodeficiency (ADA-SCID).
Multiple sclerosis (MS) patients undergoing a treatment that depletes a type of immune cell that fuels MS attacks still have a strong response to mRNA COVID-19 vaccines, a new study finds.
"The message from this study is clear — it is worthwhile for patients with MS receiving [anti-CD20] treatment to get a COVID-19 vaccine, which will prevent severe illness," said researcher E. John Whe...
Doctors for the first time have used a form of gene therapy to restore partial vision in a blind person, according to findings announced Monday.
The research team genetically altered retinal ganglion cells to become light-sensitive in a man whose vision was destroyed by retinitis pigmentosa, a genetic disorder that breaks down cells that absorb and convert light into brain signals.
Cora Oakley is a rough-and-tumble 4-year-old who loves gymnastics and outdoor activities, particularly if it involves bouncing on a trampoline.
It's hard to tell from looking at her that she was born without an immune system. Kids with this condition can acquire dangerous, life-threatening infections from day-to-day activities as simple as going to school or playing with friends.
An experimental gene therapy for Duchenne muscular dystrophy shows promise, a small study suggests.
The severe form of muscular dystrophy -- which affects about one in 3,500 males born each year in the United States -- causes muscles to progressively weaken and lose the ability to regenerate after an injury.
Muscle tissue is eventually replaced by fat and collagen. Many children wit...
It may be possible to protect Parkinson's patients' brains from further damage by turning off a "master regulator" gene, researchers report.
"One of the biggest challenges in treating Parkinson's, other than the lack of therapies that impede disease progression, is that the disease has already laid waste to significant portions of the brain by the time it is diagnosed," said researche...
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.