Publication
1/01/2002
A pilot study of in vivo liver-directed gene transfer with an adenoviral vector in partial ornithine transcarbamylase deficiency.
Raper SE, Yudkoff M, Chirmule N, Gao GP, Nunes F, Haskal ZJ, Furth EE, Propert KJ, Robinson MB, Magosin S, Simoes H, Speicher L, Hughes J, Tazelaar J, Wivel NA, Wilson JM, Batshaw ML
Human Gene Therapy
Publication
6/10/2001
Differences in the human and mouse amino-terminal leader peptides of ornithine transcarbamylase affect mitochondrial import and efficacy of adenoviral vectors.
Ye X, Zimmer KP, Brown R, Pabin C, Batshaw ML, Wilson JM, Robinson MB
Human Gene Therapy
Publication
10/01/2000
Transient depletion of CD4 lymphocyte improves efficacy of repeated administration of recombinant adenovirus in the ornithine transcarbamylase deficient sparse fur mouse.
Ye X, Robinson MB, Pabin C, Batshaw ML, Wilson JM
Gene Therapy
Publication
11/01/1999
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
Pediatric Research
Publication
9/20/1999
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
Publication
4/01/1999
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
Molecular Medicine