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September 2018

Gene Editing as a Potential Therapeutic Solution for Fuchs Endothelial Corneal Dystrophy: The Future Is Clearer

Author Affiliations
  • 1Wilmer Eye Institute, Johns Hopkins Medical Institutions, Baltimore, Maryland
  • 2Hunterian Medicine, Cambridge, Massachusetts
JAMA Ophthalmol. 2018;136(9):969-970. doi:10.1001/jamaophthalmol.2018.2324

Fuchs endothelial corneal dystrophy (FECD) is a hereditary degenerative condition that is characterized by the progression of focal excrescences in the Descemet membrane (also called the guttae) to endothelial cell loss and stromal edema. The condition remains the most common corneal dystrophy and the leading indication for corneal transplantation in developed countries.1 Though its clinical features were first described more than a century ago, advances in sequencing and genetic manipulation techniques in the past 15 years have improved the understanding of gene-based causal and pathophysiologic mechanisms, providing a pathway toward new therapeutic approaches for FECD. As the cornea is the only transparent connective tissue in the human body, any dystrophies that lead to corneal clouding can have deleterious effects on vision, which explains why surgical tissue replacement persists as the ultimate treatment for FECD. However, with recent innovations in gene editing, the easily visible and accessible nature of the cornea may even present this tissue as an archetype for implementing new therapeutic strategies elsewhere.

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