World-first: therapy to make cells young again given to a person
Nature • • health
Key Points:
- A new gene therapy trial has begun, aiming to "partially reprogram" aged cells by activating three specific genes to restore youthful cell behavior, with the first participant recently treated for glaucoma.
- The therapy targets regeneration of optic nerve neurons, which typically do not regenerate in adults, potentially reversing damage caused by glaucoma and preventing blindness.
- Safety is a major concern, as partial reprogramming carries risks such as triggering cancerous cell states; however, using the eye as a target reduces the risk of life-threatening side effects.
- The approach builds on 2020 research showing that activating these three genes in mice promoted neuron regeneration and vision restoration, with subsequent animal studies indicating no serious adverse effects.
- If successful, this trial could pave the way for broader applications of partial cellular reprogramming as a treatment for age-related diseases.