This Month in The Journal

Fuchs endothelial corneal dystrophy (FECD) is a common ocular disease caused by the expansion of a trinucleotide repeat in TCF4; greater than 50 copies of the non-coding repeat lead to increased disease risk. Like that of other repeat-expansion disorders, the pathogenicity is attributed to RNA aggregates, the sequestering of RNA splicing factors, and depletion of the encoded protein. These attributes suggest that therapies that use antisense oligonucleotides (ASOs) to target the TCF4 repeat could be effective.
Source: The American Journal of Human Genetics - Category: Genetics & Stem Cells Authors: Tags: Editors' Corner Source Type: research