top of page
Working together toward a cure

ASO Discovery
2022-26
ASO's (antisense oligonucleotides) are molecules that can bind to RNA to alter gene expression. The "discovery" phase is the first step in identifying an ASO that will work specifically to up-regulate the MYT1L gene. This work is in progress and expected to conclude within the next year.
Cre Lox Testing
2024-26
Cre Lox mice are mice bred to enable the turning on or off of a gene difference at a specific point in the mouse's lifespan. This allows scientists to study whether, and at what point in the lifespan, a gene based treatment could be effective in preventing or reversing the effects a MYT1L mutation.
bottom of page
