Dorothy Gheorghiu

Minimum Effective Dose to Achieve Biochemical Correction with Adeno-Associated Virus Vector-Mediated Gene Therapy in Mice with Pompe Disease

Human Gene Therapy · 2022 · 11 citations

Genome editing using Staphylococcus aureus Cas9 in a canine model of glycogen storage disease Ia

Molecular Therapy — Methods & Clinical Development · 2023 · 8 citations