biostudies-literatureUnknown67(2)Chen J<h4>Purpose</h4>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.<h4>Method</h4>We generated two human corneal epithelial (HCE) cell models harboring a copy of the most common disease-causing TGBFI mutations, R124C or R555W. These lines were electroporated with an ABE8e-NG-encoding mRNA and guide RNAs targeting the mutations. The resulting A•T-to-G•C editing efficiencies and off-target (OT) effects were assessed by amplicon sequencing. GFP-expressing adeno-associated viruses (AAVs) with different capsid types were transduced into HCE cells aInvestigative ophthalmology & visual science60https://www.ebi.ac.uk/biostudies/studies/S-EPMC12949465biostudies-literatureCRISPR Base Editing Correction of TGFBI Mutations in Autosomal Dominant Corneal Dystrophies.PMC12949465Hou ZPrasov LChen JEllis JBlevins BZhang YDavison CWKong DPresley WMian SIMyers MTfalseCRISPR Base Editing Correction of TGFBI Mutations in Autosomal Dominant Corneal Dystrophies.<h4>Purpose</h4>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.<h4>Method</h4>We generated two human corneal epithelial (HCE) cell models harboring a copy of the most common disease-causing TGBFI mutations, R124C or R555W. These lines were electroporated with an ABE8e-NG-encoding mRNA and guide RNAs targeting the mutations. The resulting A•T-to-G•C editing efficiencies and off-target (OT) effects were assessed by amplicon sequencing. GFP-expressing adeno-associated viruses (AAVs) with different capsid types were transduced into HCE cells a2026-01-01T00:00:00Z2026 Feb2026-07-16T23:24:44.721Z2026-07-12T03:11:18.486ZS-EPMC129494654175782410.1167/iovs.67.2.60