RARE DISEASE RESEARCH & TRAINING
A Novel Intranasal Protein-Based Therapeutic for Fragile X Syndrome
IPRD funded research by Yuan Wang, Ph.D., Zucai Suo, Ph.D., Devon Graham, Ph.D., & Deirdre McCarthy
Fragile X syndrome is a genetic condition that causes learning difficulties, developmental delays, and autism‑like behaviors. It happens because the brain lacks a protein called FMRP, which is essential for normal brain development. This project explores a new, non‑invasive treatment: delivering a working version of FMRP into the brain using tiny natural particles called extracellular vesicles (EVs), given through the nose. The team will load EVs with a safe, functional fragment of the FMRP protein, test how well brain cells take it up, and track how far it travels in the brain. If successful, this approach could become a personalized therapy that restores missing protein without using viruses or surgery.