#TGFBI #baseediting for #cornealdystrophy

#ABE8e-NG: R124C 91%, R555W 62%

Allele-specific, <0.2% indels, <0.7% bystander, <1.2% off-target

Function restored: TGFBI lysosomal mislocalization reversed

AAV1 = best corneal transduction

Dominant correction ✓

doi.org/10.1167/iovs...