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