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Items: 1 to 20 of 22

1.

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.

2.

[Gene therapy for ornithine transcarbamylase (OTC) deficient mice].

Shimada T, Saheki T.

Tanpakushitsu Kakusan Koso. 1995 Dec;40(17):2674-8. Review. Japanese. No abstract available.

PMID:
8584719
3.

Gene therapy for ornithine transcarbamylase deficiency.

Kiwaki K, Matsuda I.

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

PMID:
8677801
4.

Adeno-associated virus serotypes: vector toolkit for human gene therapy.

Wu Z, Asokan A, Samulski RJ.

Mol Ther. 2006 Sep;14(3):316-27. Epub 2006 Jul 7. Review.

5.

Clinical gene therapy using recombinant adeno-associated virus vectors.

Mueller C, Flotte TR.

Gene Ther. 2008 Jun;15(11):858-63. doi: 10.1038/gt.2008.68. Epub 2008 Apr 17. Review.

PMID:
18418415
6.

Long-term correction of murine phenylketonuria by viral gene transfer: liver versus muscle.

Thöny B.

J Inherit Metab Dis. 2010 Dec;33(6):677-80. doi: 10.1007/s10545-010-9044-3. Epub 2010 Feb 12. Review.

PMID:
20151201
7.
8.

Recent advances in liver-directed gene therapy: implications for the treatment of dyslipidemia.

Oka K, Davis AR, Chan L.

Curr Opin Lipidol. 2000 Apr;11(2):179-86. Review.

PMID:
10787180
9.

Gene therapy for human liver disease.

Raper SE, Wilson JM.

Prog Liver Dis. 1995;13:201-30. Review.

PMID:
9224503
10.

[A case of ornithine transcarbamylase deficiency presenting severe symptoms in adulthood].

Shiro Y, Yabuki S.

Rinsho Shinkeigaku. 1992 Jul;32(7):752-4. Review. Japanese.

PMID:
1291170
11.
12.

Molecular detection and correction of ornithine transcarbamylase deficiency.

Grompe M, Jones SN, Caskey CT.

Trends Genet. 1990 Oct;6(10):335-9. Review.

PMID:
2281529
13.

[Gene therapy for ornithine transcarbamylase deficiency].

Matsuda I, Kiwaki K, Komaki S, Hoshide R, Matsuura T, Endo F.

Tanpakushitsu Kakusan Koso. 1995 Dec;40(17):2759-64. Review. Japanese. No abstract available.

PMID:
8584733
14.

Ornithine transcarbamylase deficiency: pathogenesis of the cerebral disorder and new prospects for therapy.

Michalak A, Butterworth RF.

Metab Brain Dis. 1997 Sep;12(3):171-82. Review.

PMID:
9346466
15.

Mutations and polymorphisms in the human ornithine transcarbamylase gene.

Tuchman M, Jaleel N, Morizono H, Sheehy L, Lynch MG.

Hum Mutat. 2002 Feb;19(2):93-107. Review.

PMID:
11793468
16.

Adeno-associated viral vectors for clinical gene transfer studies.

Snyder RO, Francis J.

Curr Gene Ther. 2005 Jun;5(3):311-21. Review.

PMID:
15975008
17.

Primary ornithine transcarbamylase deficiency. A case report and electron microscopic study.

Aida S, Ogata T, Kamota T, Nakamura N.

Acta Pathol Jpn. 1989 Jul;39(7):451-6. Review.

PMID:
2678891
18.

Ornithine transcarbamylase deficiency: a urea cycle defect.

Gordon N.

Eur J Paediatr Neurol. 2003;7(3):115-21. Review.

PMID:
12788037
19.

[Promoter and enhancer of the ornithine transcarbamylase gene].

Nishiyori A, Takiguchi M, Mori M.

Tanpakushitsu Kakusan Koso. 1995 Dec;40(17):2606-12. Review. Japanese. No abstract available.

PMID:
8584708
20.

Sparse-fur (spf) mouse as a model of hyperammonemia: alterations in the neurotransmitter systems.

Qureshi IA, Rao KV.

Adv Exp Med Biol. 1997;420:143-58. Review. No abstract available.

PMID:
9286431

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