Developmental and Epileptic Encephalopathies (DEEs) are severe epilepsy syndromes often caused by mutations in one of the two copies of a critical gene. The goal of this project is to develop a novel Antisense Oligonucleotide (ASO) strategy to treat these syndromes by changing how these genes are processed within the cell. Dr. Felix’s team will first identify genes suitable for this approach and then generate ASOs that can boost the activity of these genes in human neurons. If successful, this approach could lead to new treatments for DEEs and help develop therapies for other brain disorders as well.
Recent Grants
