CRISPR Base Editing Correction of TGFBI Mutations in Autosomal Dominant Corneal Dystrophies.

Abstract excerpt

Purpose: Lattice and granular corneal dystrophy comprise two common TGFBI-associated autosomal dominant corneal disorders. Existing therapies are only temporizing and carry significant morbidity. Here, we develop a novel therapeutic approach using an adenine base editor (ABE) to correct common TGFBI mutations. Method: We generated two human corneal epithelial (HCE) cell models harboring a copy of the most common...

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