IPRD funded research by Yuan Wang
Fragile X syndrome (FXS) is a rare neurodevelopmental condition caused by a mutation in the fragile X messenger ribonucleoprotein 1 gene (FMR1) on the X chromosome. The syndrome is characterized by intellectual disability and behavioral problems such as social difficulties, anxiety, hyperactivity, hypersensitivity to sensory stimuli, and other autistic-like behaviors. Preclinical studies demonstrate efficacy of adeno-associated virus mediated delivery of exogenous FMR protein. However, viral transfection is associated with several challenges, including immunogenicity, low Blood Brain Barrier (BBB) crossing efficiency, off-target effects, and the need for invasive surgery. Additionally, healthy brains require different amounts of the protein at different ages. The goal of this research project is to develop a non-viral extracellular vesicle platform to deliver FMR protein in a controlled manner that can be adjusted based on the severity of phenotypes and age of the patient, as a first step toward developing personized medicine for FXS.
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