By Paul McClure A new single-dose genetic therapy has stopped the progression of two devastating neurodegenerative diseases in mice Depositphotos Researchers have developed a single-dose genetic therapy that can clear protein blockages that cause motor neurone disease, also called amyotrophic lateral sclerosis, and frontotemporal dementia, two incurable neurodegenerative diseases that eventually lead to death. In healthy...
by Ana María Rodríguez, Baylor College of Medicine Credit: CC0 Public DomainHeart failure remains the leading cause of mortality in the U.S. During a heart attack blood stops flowing into the heart. Without oxygen, part of the heart muscle dies. The heart muscle does not regenerate; instead, it replaces dead tissue with a scar made of...
by American College of Physicians Normal blood cells next to a sickle-blood cell, colored scanning electron microscope image. Credit: Wikipedia/Illustration from Anatomy & PhysiologyA modeling study comparing the cost-effectiveness of gene therapy versus common care for patients with sickle cell disease found that gene therapy is beneficial in this patient population and likely cost-effective if the...
by Robert McCoppin, Chicago Tribune Normal blood cells next to a sickle-blood cell, colored scanning electron microscope image. Credit: Wikipedia/Illustration from Anatomy & PhysiologyLyric Porter wanted her life back. Born with sickle cell disease, she had a fever at age 5 that scarred her lungs and required her to roll an oxygen tank to school....
Peer-Reviewed Publication BAYLOR COLLEGE OF MEDICINE Just like a doctor adjusts the dose of a medication to the patient’s needs, the expression of therapeutic genes, those modified in a person to treat or cure a disease via gene therapy, also needs to be maintained within a therapeutic window. Staying within the therapeutic window is important...
Nancy A. Melville November 06, 2023 PHOENIX — Children with Duchenne muscular dystrophy (DMD) treated with the only gene therapy to date to be approved for treatment of disease in the United States show sustained maintenance of motor function after 4 years, compared with untreated patients who showed significant decline over the same time period, new research...
by Catherine Caruso, Harvard Medical School Credit: Pixabay/CC0 Public DomainWhen neurobiologist David Corey showed up at a rare disease conference in 2017, he had no idea that he would enter a race against time to develop a treatment for it. The conference was for Usher syndrome type 1F. Patients with this condition have a gene mutation...
by Brigham and Women’s Hospital Glioblastoma (histology slide). Credit: Wikipedia/CC BY-SA 3.0Glioblastoma (GBM), an aggressive brain cancer, is notoriously resistant to treatment, with recurrent GBM associated with survival of less than 10 months. Immunotherapies, which mobilize the body’s immune defenses against a cancer, have not been effective for GBM, in part because the tumor’s surrounding environment...
by UC Davis Graphical Abstract. Credit: Molecular Therapy (2023). DOI: 10.1016/j.ymthe.2023.04.019 Gene therapy that induces the body to create microRNA-22 (miR-22), a naturally occurring molecule, successfully treated mice with hepatocellular carcinoma, the most common form of liver cancer. The miR-22 treatment also reduced liver inflammation and produced better survival outcomes with no observable toxicity compared...
OREGON HEALTH & SCIENCE UNIVERSITY A form of gene therapy currently used to treat Parkinson’s disease may dramatically reduce alcohol use among chronic heavy drinkers, researchers at Oregon Health & Science University and institutions across the country have found. The study in nonhuman primates showed that implanting a specific type of molecule that induces cell growth effectively...
