BioCentury
ARTICLE | Distillery Therapeutics

DLK identified as target for Fragile X syndrome

September 19, 2019 2:32 AM UTC

INDICATION: Fragile X syndrome

Inhibiting DLK, a mediator of neuronal injury responses, could treat Fragile X syndrome. In fruit flies with the disease, systemic DLK knockout or a tool compound DLK inhibitor prevented repetitive grooming behaviors in adults and reduced neurodevelopmental defects by preventing synaptic overgrowth at the larval neuromuscular junction. Next steps include testing DLK inhibition in a mouse model of Fragile X...