Researchers to test gene therapy for rare diseases with a new FDA trial protocol
Schematic of “umbrella of umbrellas” master protocol clinical trial. Credit: The American Journal of Human Genetics (2025). DOI: 10.1016/j.ajhg.2025.10.006 Earlier...
diseases
New Gene-Editing Tech Holds Promise for Treating Complex Genetic Diseases
Some genetic disorders—such as cystic fibrosis, hemophilia and Tay Sachs disease—involve many mutations in a person’s genome, often with enough...
Promising new method could treat inherited diseases | Waterloo News
An innovative method that uses modified versions of a bacterial virus effective at delivering treatments to human cells shows promise...
World-first CRISPR therapy could ‘transform’ treatment for rare genetic diseases, but key challenges lie ahead
It has been described as a revolutionary technology — and won its inventors a Nobel Prize.CRISPR gene editing, often simply...
The hunt for cell and gene therapies for eye diseases
Microscopy image showing the optic nerve in advanced glaucoma disease. Stem-cell therapy could help restore vision loss caused by degenerative...
WHO director says USAID cuts threaten progress on deadly diseases
The World Health Organization on Monday issued its starkest warning yet on the consequences of the abrupt cessation of U.S....
The Future of Gene-Editing Treatments for Rare Diseases < Yale School of Medicine
Angelman syndrome is a rare genetic disorder caused by a mutation on chromosome 15, which hinders the production of a...
Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord
Introduction Classically, the majority of medical treatments have been developed for diseases affecting large number of patients and patients with...
Europe’s medical schools to give more training on diseases linked to climate crisis | Global development
Mosquito-borne diseases such as dengue and malaria will become a bigger part of the curriculum at medical schools across Europe...
