Health Two Stem Cell Types Just Restored Vision in a First Human Trial

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Aniridia-related keratopathy (ARK) is the kind of condition that makes ophthalmologists uncomfortable. It's relentless. A PAX6 gene mutation disrupts limbal stem cells — the gatekeepers that keep the cornea clear — and conjunctival tissue quietly invades, clouding the eye like frost creeping across a window. Standard corneal transplants can't fix this. They patch the surface without addressing the broken stem cell supply underneath. For patients, that means progressive, bilateral vision loss with few effective options remaining.

Researchers at Moorfields Eye Hospital and UCL just changed that calculus.

A Two-Cell Scaffold Targets the Root Problem​

By delivering two distinct stem cell types simultaneously, RAFT-OS addresses both the surface and the structural layers conventional transplants leave untouched.

RAFT-OS — Real Architecture for 3D Tissues–Ocular Surface — is a collagen scaffold seeded with two types of donor stem cells: limbal epithelial cells (the surface-renewing layer) to rebuild the corneal surface, and stromal keratocytes (structural support cells) to stabilize the deeper tissue. Think of it like renovating a building by replacing both the facade and the load-bearing walls simultaneously, rather than just repainting over the damage.

The first-in-human trial, published July 23 in JAMA Ophthalmology, treated nine adults with advanced ARK. Key findings:

 
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