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 8/14/2000

Genotype spectrum of ornithine transcarbamylase deficiency: correlation with the clinical and biochemical phenotype.

McCullough BA, Yudkoff M, Batshaw ML, Wilson JM, Raper SE, Tuchman M

American Journal Of Medical Genetics

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 1/01/1998

Developing adenoviral-mediated in vivo gene therapy for ornithine transcarbamylase deficiency.

Raper SE, Wilson JM, Yudkoff M, Robinson MB, Ye X, Batshaw ML

Journal Of Inherited Metabolic Disease

Publication 11/01/1996

In vivo nitrogen metabolism in ornithine transcarbamylase deficiency.

Yudkoff M, Daikhin Y, Nissim I, Jawad A, Wilson J, Batshaw M

The Journal Of Clinical Investigation