Colleen Le

Gene therapy for guanidinoacetate methyltransferase deficiency restores cerebral and myocardial creatine while resolving behavioral abnormalities

Molecular Therapy — Methods & Clinical Development · 2022 · 10 citations

CRISPR-Mediated Genomic Addition to CPS1 Deficient iPSCs is Insufficient to Restore Nitrogen Homeostasis

The Yale Journal of Biology and Medicine · 2021 · 10 citations