Michael P. Hughes

This study investigates the impact of LRRK2 G2019S mutation on Wnt and NFAT signaling in Parkinson's disease models, finding significant dysregulation in specific brain regions.

The paper develops a method using adeno-associated virus vectors to generate somatic-transgenic mice for non-invasive monitoring of signaling pathways in disease models.

This study shows that neonatal brain-directed gene therapy using AAV9-CLN6 significantly improves survival, motor function, and reduces neuropathology in a mouse model of CLN6 Batten disease.

This paper develops a non-integrating lentiviral gene therapy for refractory focal epilepsy, showing reduced seizure frequency in a rat model.

This paper demonstrates that gene therapy using AAV8 vector corrects urea cycle and citrulline-NO cycle defects in ASL-deficient mice, reducing neuronal disease and improving behavior.