A new gene therapy turns glial cells — abundant support cells in the brain — into neurons, repairing damage that results from stroke and significantly improving motor function in mice. A paper describing the new therapy, which uses the NeuroD1 gene, appears online in the journal Molecular Therapy. Once further developed, this NeuroD1-based gene therapy potentially
An FDA-approved gene therapy for Leber congenital amaurosis, an inherited vision disorder with a childhood onset and progressive nature, has improved patients’ sight. But new research underscores the importance of further investigation to halt the progression of the disorder. Nearly two decades ago, a gene therapy restored vision to Lancelot, a Briard dog who was
In pair of studies, UCLA researchers describe new ways to treat a metabolic liver disorder Source UCLA Newsroom Most babies who are born with arginase deficiency — a rare genetic disease that leads to the accumulation of the amino acid arginine in the blood — don’t have symptoms at first. By the time they’re toddlers,
Gene therapies constitute a new concept of transformative therapies, administered once in a lifetime. The value assessment of these innovative therapies constitutes a challenge for health technology assessment (HTA) bodies. The HTA reports for all seven gene therapies that have to date been granted a market authorization in the European Union (EU) and/or the United
Increased Time to Hypoglycemia and Reduction in Cornstarch Use in Cohorts 1 and 2; Improvement of Additional Key Metabolic Measures Observed Cohort 1 Patients Continue to Demonstrate Long-Term, Durable Responses Company to Enroll Three Patients in Expansion Cohort to Confirm 6.0 × 10^12 GC/kg Dose as Optimal Dose for Phase 3 Study Ultragenyx Pharmaceutical, a
Breaking down how the gene editing technology is being used, for the first time in the United States, to treat patients with severe medical conditions Source Smithsonian.com Last fall, the birth of genetically edited twin girls in China—the world’s first “designer babies”—prompted an immediate outcry in the medical science community. The change to the twins’ genomes,
It was a crucial moment when NHS England and the National Institute for Health and Care Excellence (NICE) approved Chimeric Antigen Receptor T-cell (CAR-T) therapies for use on the NHS last year. CAR-T therapy uses a patient’s own white blood cells to fight cancer – England became one of the first countries in the world to make
Early clinical trials of a gene therapy for aromatic amino acid decarboxylase (AADC) deficiency have yielded encouraging results for its safety and efficacy, a study highlights. But researchers call attention to the importance of a timely diagnosis for treatment success, as only early interventions could fully prevent long-term brain damage and other outcomes. Their study, “Aromatic amino
A promising new treatment for cancer patients has received approval from the Centers for Medicare and Medicaid Services, paving the way for more patients to get the treatment known as CAR T-cell therapy. While this decision will likely expand access, particularly for some lymphoma and leukemia patients, treatment remains prohibitively expensive, prompting some patients to opt for experimental rather
Research led by Dr. Krystof Bankiewicz, who recently joined The Ohio State University College of Medicine, shows that gene replacement therapy for Niemann-Pick type A disease is safe for use in nonhuman primates and has therapeutic effects in mice. These research findings are published online in the journal Science Translational Medicine. Prior to joining Ohio State as a professor
