The first person has been treated with a highly anticipated new gene therapy that aims to turn back the clock on ageing cells. The trial targets retinal cells, with the hope that encouraging them to behave as if they were young again could improve sight in the affected patients.
How the Therapy Works
According to science editor Ian Sample, the therapy involves delivering genes that encode for reprogramming factors, which can reset the epigenetic markers on DNA to a more youthful state. This approach, known as cellular rejuvenation, has shown promise in animal studies, reversing signs of ageing in mice and extending their lifespan.
Potential Impact and Risks
Paul Knoepfler, professor of cell biology and human anatomy at the University of California, Davis, warns that the therapy carries risks, including the potential for uncontrolled cell growth leading to cancer. However, the trial is designed with safety measures, such as using a temporary expression system that turns off after a few weeks.
If the therapy proves safe and effective in the eye, it could pave the way for treatments for other age-related diseases, such as Alzheimer's, heart disease, and arthritis. The field of cellular rejuvenation is rapidly advancing, with several other companies developing similar approaches.
Next Steps
The current trial is a phase 1 safety study, involving a small number of patients. Results are expected within a year. If successful, larger trials will be needed to confirm efficacy and safety before the therapy can become widely available.
