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Items: 1 to 20 of 102

1.

Recombinant adeno-associated virus integration sites in murine liver after ornithine transcarbamylase gene correction.

Zhong L, Malani N, Li M, Brady T, Xie J, Bell P, Li S, Jones H, Wilson JM, Flotte TR, Bushman FD, Gao G.

Hum Gene Ther. 2013 May;24(5):520-5. doi: 10.1089/hum.2012.112.

2.

DLK1-DIO3 genomic imprinted microRNA cluster at 14q32.2 defines a stemlike subtype of hepatocellular carcinoma associated with poor survival.

Luk JM, Burchard J, Zhang C, Liu AM, Wong KF, Shek FH, Lee NP, Fan ST, Poon RT, Ivanovska I, Philippar U, Cleary MA, Buser CA, Shaw PM, Lee CN, Tenen DG, Dai H, Mao M.

J Biol Chem. 2011 Sep 2;286(35):30706-13. doi: 10.1074/jbc.M111.229831. Epub 2011 Jul 7.

3.

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
4.

AAV vector integration sites in mouse hepatocellular carcinoma.

Donsante A, Miller DG, Li Y, Vogler C, Brunt EM, Russell DW, Sands MS.

Science. 2007 Jul 27;317(5837):477.

5.

Large-scale molecular characterization of adeno-associated virus vector integration in mouse liver.

Nakai H, Wu X, Fuess S, Storm TA, Munroe D, Montini E, Burgess SM, Grompe M, Kay MA.

J Virol. 2005 Mar;79(6):3606-14.

6.

Frequency and spectrum of genomic integration of recombinant adeno-associated virus serotype 8 vector in neonatal mouse liver.

Inagaki K, Piao C, Kotchey NM, Wu X, Nakai H.

J Virol. 2008 Oct;82(19):9513-24. doi: 10.1128/JVI.01001-08. Epub 2008 Jul 9.

7.

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
8.

Recombinant adeno-associated virus-mediated correction of lysosomal storage within the central nervous system of the adult mucopolysaccharidosis type VII mouse.

Sferra TJ, Qu G, McNeely D, Rennard R, Clark KR, Lo WD, Johnson PR.

Hum Gene Ther. 2000 Mar 1;11(4):507-19.

PMID:
10724030
9.

Assessing the potential for AAV vector genotoxicity in a murine model.

Li H, Malani N, Hamilton SR, Schlachterman A, Bussadori G, Edmonson SE, Shah R, Arruda VR, Mingozzi F, Wright JF, Bushman FD, High KA.

Blood. 2011 Mar 24;117(12):3311-9. doi: 10.1182/blood-2010-08-302729. Epub 2010 Nov 24. Erratum in: Blood. 2011 Jun 16;117(24):6739.

10.

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.

11.

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.

PMID:
16682254
12.

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.

13.

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.

14.

Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors.

Donsante A, Vogler C, Muzyczka N, Crawford JM, Barker J, Flotte T, Campbell-Thompson M, Daly T, Sands MS.

Gene Ther. 2001 Sep;8(17):1343-6.

15.
16.

Vector sequences are not detected in tumor tissue from research subjects with ornithine transcarbamylase deficiency who previously received adenovirus gene transfer.

Zhong L, Li S, Li M, Xie J, Zhang Y, Lee B, Batshaw ML, Wilson JM, Gao G.

Hum Gene Ther. 2013 Sep;24(9):814-9. doi: 10.1089/hum.2013.118.

17.
18.

Selection and evaluation of clinically relevant AAV variants in a xenograft liver model.

Lisowski L, Dane AP, Chu K, Zhang Y, Cunningham SC, Wilson EM, Nygaard S, Grompe M, Alexander IE, Kay MA.

Nature. 2014 Feb 20;506(7488):382-6. doi: 10.1038/nature12875. Epub 2013 Dec 25.

19.

Patterns of scAAV vector insertion associated with oncogenic events in a mouse model for genotoxicity.

Rosas LE, Grieves JL, Zaraspe K, La Perle KM, Fu H, McCarty DM.

Mol Ther. 2012 Nov;20(11):2098-110. doi: 10.1038/mt.2012.197. Epub 2012 Sep 18.

20.

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
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