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Results: 1 to 20 of 108

1.

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
[PubMed - indexed for MEDLINE]
2.

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
[PubMed - indexed for MEDLINE]
3.

Differences in the human and mouse amino-terminal leader peptides of ornithine transcarbamylase affect mitochondrial import and efficacy of adenoviral vectors.

Ye X, Zimmer KP, Brown R, Pabin C, Batshaw ML, Wilson JM, Robinson MB.

Hum Gene Ther. 2001 Jun 10;12(9):1035-46.

PMID:
11399226
[PubMed - indexed for MEDLINE]
4.

Patient selection may affect gene therapy success. Dominant negative effects observed for ornithine transcarbamylase in mouse and human hepatocytes.

Morsy MA, Zhao JZ, Ngo TT, Warman AW, O'Brien WE, Graham FL, Caskey CT.

J Clin Invest. 1996 Feb 1;97(3):826-32.

PMID:
8609240
[PubMed - indexed for MEDLINE]
Free PMC Article
5.

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.

PMID:
16677864
[PubMed - indexed for MEDLINE]
6.

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.

PMID:
19384294
[PubMed - indexed for MEDLINE]
Free PMC Article
7.

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.

PMID:
8631974
[PubMed - indexed for MEDLINE]
Free Article
8.

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.

PMID:
21386824
[PubMed - indexed for MEDLINE]
Free PMC Article
9.

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
[PubMed - indexed for MEDLINE]
10.
11.

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.

Hum Gene Ther. 2002 Jan 1;13(1):163-75.

PMID:
11779420
[PubMed - indexed for MEDLINE]
12.

Efficient adenoviral-mediated ornithine transcarbamylase expression in deficient mouse and human hepatocytes.

Morsy MA, Alford EL, Bett A, Graham FL, Caskey CT.

J Clin Invest. 1993 Sep;92(3):1580-6.

PMID:
8376608
[PubMed - indexed for MEDLINE]
Free PMC Article
13.

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
[PubMed - indexed for MEDLINE]
14.

Expression analysis of two mutant human ornithine transcarbamylases in COS-7 cells.

Kogo T, Satoh Y, Kanazawa M, Yamamoto S, Takayanagi M, Ohtake A, Mori M, Niimi H.

J Hum Genet. 1998;43(1):54-8.

PMID:
9609999
[PubMed - indexed for MEDLINE]
15.

Gene therapy for ornithine transcarbamylase deficiency.

Kiwaki K, Matsuda I.

Acta Paediatr Jpn. 1996 Apr;38(2):189-92. Review.

PMID:
8677801
[PubMed - indexed for MEDLINE]
16.

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
[PubMed - indexed for MEDLINE]
17.
18.

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.

PMID:
22133298
[PubMed - indexed for MEDLINE]
Free PMC Article
19.

Long-term correction of ornithine transcarbamylase deficiency by WPRE-mediated overexpression using a helper-dependent adenovirus.

Mian A, McCormack WM Jr, Mane V, Kleppe S, Ng P, Finegold M, O'Brien WE, Rodgers JR, Beaudet AL, Lee B.

Mol Ther. 2004 Sep;10(3):492-9.

PMID:
15336649
[PubMed - indexed for MEDLINE]
20.

Expression of wild-type and mutant human ornithine transcarbamylase genes in Chinese hamster ovary cells and lack of dominant negative effect of R141Q and R40H mutants.

Augustin L, Mavinakere M, Morizono H, Tuchman M.

Pediatr Res. 2000 Dec;48(6):842-6.

PMID:
11102556
[PubMed - indexed for MEDLINE]

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