What is it about?
Corneal dystrophy causes vision loss and is associated with protein clumps that form and cloud the cornea. Using electron microscopy, we obtained an image of these protein clumps at near-atomic detail. This allowed us to design molecules that target a structural weak point and block further protein clumping.
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Why is it important?
This is the first molecular image of the protein clumps associated with this disease, revealing how a genetic mutation can drive their formation. This high-resolution image identifies structural targets that can be exploited to design drugs against protein clumping — a step toward future therapies for this blinding condition.
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This page is a summary of: Cryo-EM structure of TGFBIp fibrils driven by a corneal dystrophy–linked mutation enables design of peptide inhibitors of aggregation, Proceedings of the National Academy of Sciences, September 2026, Proceedings of the National Academy of Sciences,
DOI: 10.1073/pnas.2607937123.
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