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Scientists improve vision in rare hereditary eye disease with stem cell scaffold

1 min
Scientists improve vision in rare hereditary eye disease with stem cell scaffold

This digest was compiled by AI from multiple sources — links to the originals are below.

Researchers report the first successful use of a collagen scaffold carrying two types of stem cells to treat aniridia-related keratopathy, a rare hereditary eye disease previously considered incurable. In a nine-patient study, treated eyes improved after three months, and after one year patients on average read 24 more symbols on a standard visual acuity test.

Key Facts

  • The therapy is the first successful worldwide use of a collagen scaffold with two types of stem cells for aniridia-related keratopathy.
  • Aniridia-related keratopathy develops from a mutation in the PAX6 gene that causes corneal stem cell deficiency and progressive vision loss.
  • Nine patients took part in the study; only one eye in each patient was treated, while the other eye served as a comparison.
  • After three months, the surface of the treated eye had improved significantly.
  • After one year, patients on average read 24 more symbols on a standard visual acuity test.

Aniridia-Related Keratopathy

Aniridia-related keratopathy develops from a mutation in the PAX6 gene that causes a deficiency of corneal stem cells. The corneal surface then loses transparency and is progressively covered by conjunctiva, causing vision to worsen. Before this study, no effective treatment existed for the condition.

Stem Cell Therapy Results

Researchers created a collagen scaffold loaded with two types of stem cells and implanted it on the surface of the affected eye. The study enrolled nine patients, treating only one eye in each patient and using the other eye for comparison. After three months, the surface of the treated eye had improved significantly. After one year, patients read on average 24 more symbols on a standard visual acuity test. The authors describe this as the first successful use worldwide of a collagen scaffold with two stem cell types for the disease.

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