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