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Items: 1 to 20 of 131

1.

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.

PMID:
24108150
2.

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.

3.

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.

4.

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.

5.

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.

6.

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.

7.

Modeling correction of severe urea cycle defects in the growing murine liver using a hybrid recombinant adeno-associated virus/piggyBac transposase gene delivery system.

Cunningham SC, Siew SM, Hallwirth CV, Bolitho C, Sasaki N, Garg G, Michael IP, Hetherington NA, Carpenter K, de Alencastro G, Nagy A, Alexander IE.

Hepatology. 2015 Aug;62(2):417-28. doi: 10.1002/hep.27842. Epub 2015 May 23.

PMID:
26011400
8.

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.

9.

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.

PMID:
8860834
10.

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice.

Yang Y, Wang L, Bell P, McMenamin D, He Z, White J, Yu H, Xu C, Morizono H, Musunuru K, Batshaw ML, Wilson JM.

Nat Biotechnol. 2016 Mar;34(3):334-8. doi: 10.1038/nbt.3469. Epub 2016 Feb 1.

11.

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.

PMID:
19357713
13.
14.

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.

15.

Retroviral-mediated gene transfer of human ornithine transcarbamylase into primary hepatocytes of spf and spf-ash mice.

Grompe M, Jones SN, Loulseged H, Caskey CT.

Hum Gene Ther. 1992 Feb;3(1):35-44.

PMID:
1562638
16.

Evaluation of the transfer and expression in mice of an enzyme-encoding gene using a human adenovirus vector.

Stratford-Perricaudet LD, Levrero M, Chasse JF, Perricaudet M, Briand P.

Hum Gene Ther. 1990 Fall;1(3):241-56.

PMID:
2081192
17.
18.

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.

PMID:
7999674
19.

Importance of ornithine transcarbamylase (OTC) deficiency in small intestine for urinary orotic acid excretion: analysis of OTC-deficient spf-ash mice with OTC transgene.

Saheki T, Mori K, Kobayashi K, Horiuchi M, Shige T, Obara T, Suzuki S, Mori M, Yamamura K.

Biochim Biophys Acta. 1995 Jan 25;1270(1):87-93.

PMID:
7827141
20.

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.

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