Patrick F. Finn

This paper demonstrates the use of LNP-delivered prime editors to correct the PAH R408W mutation in PKU models, achieving ~50% correction efficiency.

This study shows that clodronate liposomes enhance lentiviral liver transduction but not AAV or LNP-mRNA in mice.

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