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Long-term correction of ammonia metabolism and prolonged survival in ornithine transcarbamylase-deficient mice following liver-directed treatment with adeno-associated viral vectors.

Moscioni D, Morizono H, McCarter RJ, Stern A, Cabrera-Luque J, Hoang A, Sanmiguel J, Wu D, Bell P, Gao GP, Raper SE, Wilson JM, Batshaw ML.

Mol Ther. 2006 Jul;14(1):25-33. Epub 2006 May 3.


Analysis of tumors arising in male B6C3F1 mice with and without AAV vector delivery to liver.

Bell P, Moscioni AD, McCarter RJ, Wu D, Gao G, Hoang A, Sanmiguel JC, Sun X, Wivel NA, Raper SE, Furth EE, Batshaw ML, Wilson JM.

Mol Ther. 2006 Jul;14(1):34-44. Epub 2006 May 6.


Prolonged metabolic correction in adult ornithine transcarbamylase-deficient mice with adenoviral vectors.

Ye X, Robinson MB, Batshaw ML, Furth EE, Smith I, Wilson JM.

J Biol Chem. 1996 Feb 16;271(7):3639-46.


AAV2/8-mediated correction of OTC deficiency is robust in adult but not neonatal Spf(ash) mice.

Cunningham SC, Spinoulas A, Carpenter KH, Wilcken B, Kuchel PW, Alexander IE.

Mol Ther. 2009 Aug;17(8):1340-6. doi: 10.1038/mt.2009.88. Epub 2009 Apr 21.


AAV-encoded OTC activity persisting to adulthood following delivery to newborn spf(ash) mice is insufficient to prevent shRNA-induced hyperammonaemia.

Cunningham SC, Kok CY, Spinoulas A, Carpenter KH, Alexander IE.

Gene Ther. 2013 Dec;20(12):1184-7. doi: 10.1038/gt.2013.51. Epub 2013 Oct 10.


Preclinical evaluation of a clinical candidate AAV8 vector for ornithine transcarbamylase (OTC) deficiency reveals functional enzyme from each persisting vector genome.

Wang L, Morizono H, Lin J, Bell P, Jones D, McMenamin D, Yu H, Batshaw ML, Wilson JM.

Mol Genet Metab. 2012 Feb;105(2):203-11. doi: 10.1016/j.ymgme.2011.10.020. Epub 2011 Nov 7.


Transient hyperammonemia during aging in ornithine transcarbamylase-deficient, sparse-fur mice.

Gushiken T, Yoshimura N, Saheki T.

Biochem Int. 1985 Nov;11(5):637-43.


Induction and prevention of severe hyperammonemia in the spfash mouse model of ornithine transcarbamylase deficiency using shRNA and rAAV-mediated gene delivery.

Cunningham SC, Kok CY, Dane AP, Carpenter K, Kizana E, Kuchel PW, Alexander IE.

Mol Ther. 2011 May;19(5):854-9. doi: 10.1038/mt.2011.32. Epub 2011 Mar 8.


Correction of ornithine transcarbamylase deficiency in adult spf(ash) mice and in OTC-deficient human hepatocytes with recombinant adenoviruses bearing the CAG promoter.

Kiwaki K, Kanegae Y, Saito I, Komaki S, Nakamura K, Miyazaki JI, Endo F, Matsuda I.

Hum Gene Ther. 1996 May 1;7(7):821-30.


Sustained correction of OTC deficiency in spf( ash) mice using optimized self-complementary AAV2/8 vectors.

Wang L, Wang H, Morizono H, Bell P, Jones D, Lin J, McMenamin D, Yu H, Batshaw ML, Wilson JM.

Gene Ther. 2012 Apr;19(4):404-10. doi: 10.1038/gt.2011.111. Epub 2011 Aug 18.


In vivo assessment of mutations in OTC for dominant-negative effects following rAAV2/8-mediated gene delivery to the mouse liver.

Ginn SL, Cunningham SC, Zheng M, Spinoulas A, Carpenter KH, Alexander IE.

Gene Ther. 2009 Jun;16(6):820-3. doi: 10.1038/gt.2009.38. Epub 2009 Apr 9.


Gene therapy with novel adeno-associated virus vectors substantially diminishes atherosclerosis in a murine model of familial hypercholesterolemia.

Lebherz C, Gao G, Louboutin JP, Millar J, Rader D, Wilson JM.

J Gene Med. 2004 Jun;6(6):663-72.


Normalization of hair growth in sparse fur-abnormal skin and hair (SPF-ASH) mice by introduction of the rat ornithine transcarbamylase (OTC) gene.

Shimada T, Tashiro M, Kanzaki T, Noda T, Murakami T, Takiguchi M, Mori M, Yamamura K, Saheki T.

J Dermatol Sci. 1994 Jul;7 Suppl:S27-32.


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

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

J Inherit Metab Dis. 1998;21 Suppl 1:119-37.


No increase of ornithine concentration in the liver of ornithine transcarbamylase-deficient spf-ash mice following intraperitoneal injection of ammonium chloride.

Saheki T, Fukushige T, Kakinoki H, Kobayashi K.

Contrib Nephrol. 1997;121:109-16. No abstract available.


Interaction between murine spf-ash mutation and genetic background yields different metabolic phenotypes.

Marini JC, Erez A, Castillo L, Lee B.

Am J Physiol Endocrinol Metab. 2007 Dec;293(6):E1764-71. Epub 2007 Oct 9.


Complete prevention of atherosclerosis in apoE-deficient mice by hepatic human apoE gene transfer with adeno-associated virus serotypes 7 and 8.

Kitajima K, Marchadier DH, Miller GC, Gao GP, Wilson JM, Rader DJ.

Arterioscler Thromb Vasc Biol. 2006 Aug;26(8):1852-7. Epub 2006 Jun 8.


Phenotypic correction of ornithine transcarbamylase deficiency using low dose helper-dependent adenoviral vectors.

Brunetti-Pierri N, Clarke C, Mane V, Palmer DJ, Lanpher B, Sun Q, O'Brien W, Lee B.

J Gene Med. 2008 Aug;10(8):890-6. doi: 10.1002/jgm.1218.

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