“Scientists in the UK have successfully used gene therapy to restore some vision to legally blind children with an inherited retinal condition. All 11 children in the clinical trial saw improvements within weeks of a single surgical treatment.” ~ learn […]
This article in Scientific American recounts the history of gene therapy, its promise, and its multiple failures. There is continued hope as researchers find new ways to minimize risks. A quote from one gene therapy company’s chief medical officer: “I […]
“This weekend brought some really significant news in the long-running effort to use gene editing to treat human disease. As most readers will have heard, Boston Children’s Hospital and a Vertex/CRISPR effort both published papers in the NEJM addressing sickle-cell […]
“Scientists in Australia have made an exciting breakthrough in Alzheimer’s research, demonstrating what they describe as the first gene-therapy-based approach for treating advanced forms of the disease.” | learn more
“U.S. health officials … approved the nation’s first gene therapy for an inherited disease, a treatment that improves the sight of patients with a rare form of blindness.” | learn more
The FDA doesn’t need to listen to this panel, but they usually do. Scientists remove T-cells, genetically modify them to attack cancer, then put them back in the bodies of leukemia patients. This would be the first time the FDA […]