Psychoeducational profile of the 22q11.2 microdeletion: A complex pattern von Moss, Edward M., Batshaw, Mark L., Solot, Cynthia B., Gerdes, Marsha, McDonald-McGinn, Donna M., Driscoll, Deborah A., Emanuel, Beverly S., Zackai, Elaine H., Wang, Paul P.

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Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer von Raper, Steven E, Chirmule, Narendra, Lee, Frank S, Wivel, Nelson A, Bagg, Adam, Gao, Guang-ping, Wilson, James M, Batshaw, Mark L

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Long-term gene transfer to mouse fetuses with recombinant adenovirus and adeno-associated virus (AAV) vectors von MITCHELL, M, JEREBTSOVA, M, BATSHAW, M. L, NEWMAN, K, YEL, X

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Sustained correction of OTC deficiency in spf( ash) mice using optimized self-complementary AAV2/8 vectors von Wang, L, Wang, H, Morizono, H, Bell, P, Jones, D, Lin, J, McMenamin, D, Yu, H, Batshaw, M L, Wilson, J M

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Alternative pathway therapy for urea cycle disorders: Twenty years later von Batshaw, Mark L., MacArthur, Robert B., Tuchman, Mendel

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A pilot study of in vivo liver-directed gene transfer with an adenoviral vector in partial ornithine transcarbamylase deficiency von Raper, Steven E, Yudkoff, Marc, Chirmule, Narendra, Gao, Guang-Ping, Nunes, Fred, Haskal, Ziv J, Furth, Emma E, Propert, Kathleen J, Robinson, Michael B, Magosin, Susan, Simoes, Heather, Speicher, Lisa, Hughes, Joseph, Tazelaar, John, Wivel, Nelson A, Wilson, James M, Batshaw, Mark L

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Correction of argininosuccinate synthetase (AS) deficiency in a murine model of citrullinemia with recombinant adenovirus carrying human AS cDNA von YE, X, WHITEMAN, B, JEREBTSOVA, M, BATSHAW, M. L

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Transient depletion of CD4 lymphocyte improves efficacy of repeated administration of recombinant adenovirus in the ornithine transcarbamylase deficient sparse fur mouse von YE, X, ROBINSON, M. B, PABIN, C, BATSHAW, M. L, WILSON, J. M

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Cognitive outcome in urea cycle disorders von Gropman, Andrea L, Batshaw, Mark L

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Inborn errors of urea synthesis von Batshaw, Mark L.

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Sustained correction of OTC deficiency in spfash mice using optimized self-complementary AAV2/8 vectors von Wang, L, Wang, H, Morizono, H, Bell, P, Jones, D, Lin, J, McMenamin, D, Yu, H, Batshaw, M L, Wilson, J M

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Developing adenoviral‐mediated in vivo gene therapy for ornithine transcarbamylase deficiency von Raper, S. E., Wilson, J. M., Yudkoff, M., Robinson, M. B., Ye, X., Batshaw, M. L.

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Recombinant adenovirus gene transfer in adults with partial ornithine transcarbamylase deficiency (OTCD) von Batshaw, M L, Wilson, J M, Raper, S, Yudkoff, M, Robinson, M B

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Quinolinic acid in children with congenital hyperammonemia von Batshaw, Mark L., Robinson, Michael B., Hyland, Keith, Djali, Sina, Heyes, Melvyn P.

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In vivo measurement of ureagenesis with stable isotopes von Yudkoff, M., Daikhin, Y., Ye, X., Wilson, J. M., Batshaw, M. L.

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Differences in the human and mouse amino-terminal leader peptides of ornithine transcarbamylase affect mitochondrial import and efficacy of adenoviral vectors von XUEHAI YE, ZIMMER, Klaus-Peter, BROWN, Rebecca, PABIN, Carol, BATSHAW, Mark L, WILSON, James M, ROBINSON, Michael B

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Correction of ureagenesis after gene transfer in an animal model and after liver transplantation in humans with ornithine transcarbamylase deficiency von BATSHAW, M. L, ROBINSON, M. B, XUEHAI YE, PABIN, C, DAIKHIN, Y, BURTON, B. K, WILSON, J. M, YUDKOFF, M

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Prolonged Metabolic Correction in Adult Ornithine Transcarbamylase-deficient Mice with Adenoviral Vectors (∗) von Ye, Xuehai, Robinson, Michael B., Batshaw, Mark L., Furth, Emma E., Smith, Ian, Wilson, James M.

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Risk of serious illness in heterozygotes for ornithine transcarbamylase deficiency von BATSHAW, M. L, MSALL, M, BEAUDET, A. L, TROJAK, J

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Deficiency of the iron-sulfur clusters of mitochondrial reduced nicotinamide-adenine dinucleotide-ubiquinone oxidoreductase (complex I) in an infant with congenital lactic acidosis von MOREADITH, R. W, BATSHAW, M. L, OHNISHI, T, KERR, D, KNOX, B, JACKSON, D, HRUBAN, R, OLSON, J, BALTAZAR REYNAFARJE, LEHNINGER, A. L

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