A major scientific breakthrough in gene therapy has been recognized with one of the most prestigious scientific awards, often referred to as the “Science Oscar.”
According to The Guardian, the prize was awarded to a research team that developed a treatment method capable of partially restoring vision in patients with severe inherited forms of blindness.
The laureates include scientists who worked on a therapy called Luxturna—the first approved gene therapy targeting an inherited retinal disease caused by mutations in the RPE65 gene. The research was conducted over more than 20 years with the participation of specialists from the University of Pennsylvania and affiliated clinical centers.
The method involves delivering a functional copy of the defective gene into retinal cells using a viral vector. This allows partial restoration of visual function in patients who had previously lost most or nearly all of their sight. Clinical trials showed that some patients were able, after treatment, to recognize faces, distinguish shapes, and perceive movement for the first time.
The significance of the development lies in the fact that it became the first commercially approved gene therapy for inherited blindness and laid the foundation for further advances in ophthalmology. Experts estimate that this approach has already inspired the creation of more than a dozen new gene therapies for various diseases.
Researchers at the University of Pennsylvania involved in the development emphasize that a key factor in the success was years of collaboration between molecular biologists, ophthalmologists, and clinical trial specialists. According to them, it was the combination of fundamental science and applied medicine that made it possible to bring the technology into real-world use.
Experts underline that the award reflects the growing importance of genetic medicine, which is gradually moving from the experimental stage to real treatment of previously incurable diseases.
Follow NEWS.am Medicine on Facebook and Twitter