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...
by University of Michigan Credit: National Institutes of Health Research from experts at Michigan Medicine, the Children’s Hospital of Philadelphia and Penn Medicine is breaking ground on new ways of treating blood disorders, such as sickle cell anemia, through gene therapy. The study is published in the journal Science. To cure blood disorders, patients must undergo high dose chemotherapy...
by University of Bristol Image [left to right]: Professor Moin Saleem, Dr Wen Ding [Academic Clinical Lecturer in Paediatric Renal Medicine] and Professor Gavin Welch [Professor of Renal Cell Biology] from the University of Bristol’s Medical School Credit: Kidney Research UK Researchers at the University of Bristol have made a remarkable step forward in finding a potential cure for...
