Joanne Ng

Neuronal targeted AAV Micro-Dystrophin restores neurobehavioural co-morbidities, grip strength and motor coordination in mdx52 mouse model of Duchenne Muscular Dystrophy

This study evaluates neuronal-targeted AAV micro-dystrophin therapy in mdx52 mice, showing IV delivery improves neurobehavioural, motor, and neuromuscular deficits more effectively than ICV delivery.

Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors

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.

Gene therapy in argininosuccinic aciduria

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.