COMPOSITIONS AND METHODS FOR TREATMENT OF DISEASES ASSOCIATED WITH TRINUCLEOTIDE REPEATS IN TRANSCRIPTION FACTOR FOUR.

摘要

This application relates to compositions and methods for excising trinucleotide repeats (TNRs) contained within intron 3 of TCF4, such as is seen in subjects having Fuchs endothelial corneal dystrophy (FECD), PSC, and Schizophrenia. Compositions comprising guide sequences targeting the alpha 2 subunit of collagen VIII are also disclosed for treatment of mutations therein that may contribute to FECD.