Article
Biallelic rescue of CTG18.1 in two Fuchs endothelial corneal dystrophy-derived iPSC lines (SCTCi047-A-2, SCTCi046-A-2) following a two-step gene editing strategy.
Stem cell research - 1 Sept 2026
Landi Elisa, van Beusekom Ellen, Ben-Dor Shifra, Albert Silvia, Dickman Mor M, LaPointe Vanessa L S, van Bokhoven Hans
Abstract excerpt
Fuchs endothelial corneal dystrophy (FECD) is an age-related condition distinguished by the degeneration of the corneal endothelium. An intronic CTG18.1 repeat in the transcription factor 4 (TCF4) gene has been associated with a 78-fold increased risk of developing the disease when at least one copy of the CTG18.1 expands above 50 repeats. Employing patient-derived material, we applied a dual CRISPR/Cas9-mediated...
Read the complete abstract on PubMedTopics
- Humans
- Induced Pluripotent Stem Cells
- Fuchs' Endothelial Dystrophy
- Gene Editing
- Cell Line
