Correction of ureagenesis after gene transfer in an animal model and after liver transplantation in humans with ornithine transcarbamylase deficiency.
Batshaw ML, Robinson MB, Ye X, Pabin C, Daikhin Y, Burton BK, Wilson JM, Yudkoff M
Recombinant adenovirus gene transfer in adults with partial ornithine transcarbamylase deficiency (OTCD).
Batshaw ML, Wilson JM, Raper S, Yudkoff M, Robinson MB
Human Gene Therapy
Efficient mitochondrial import of newly synthesized ornithine transcarbamylase (OTC) and correction of secondary metabolic alterations in spf(ash) mice following gene therapy of OTC deficiency.
Zimmer KP, Bendiks M, Mori M, Kominami E, Robinson MB, Ye X, Wilson JM