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
Long-term regulated expression of growth hormone in mice after intramuscular gene transfer.
Rivera VM, Ye X, Courage NL, Sachar J, Cerasoli F Jr, Wilson JM, Gilman M
Proceedings Of The National Academy Of Sciences Of The United States Of America
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
Regulated delivery of therapeutic proteins after in vivo somatic cell gene transfer.
Ye X, Rivera VM, Zoltick P, Cerasoli F Jr, Schnell MA, Gao G, Hughes JV, Gilman M, Wilson JM