Youssef Khalil

This paper develops a pipeline to identify and evaluate novel manganese chelators using zebrafish and mouse models, finding that H3PyC3A and Me3PyC3A show improved manganese mobilization and reduced accumulation.

This study shows that mRNA therapy restores ureagenesis and glutathione metabolism in ASL-deficient mice, correcting liver disease and neurocognitive impairments.

This study shows that activating autophagy via Beclin-1 improves biochemical and clinical outcomes in two types of urea cycle disorders.