Tetradecanoylphorbol-13-acetate (TPA) significantly increases AAV2/5 transduction of human neuronal cells in vitro

Experimental Eye Research

Volumn 97, Issue 1, 2012, Pages 148-153

  You, Qisheng ^a,d   Brown, Laurence A ^a   McClements, Michelle ^a   Hankins, Mark W ^a   MacLaren, Robert E ^a,b,c  

^a UNIVERSITY OF OXFORD   (United Kingdom)

^b MOORFIELDS EYE HOSPITAL   (United Kingdom)

^c OXFORD UNIVERSITY HOSPITALS NHS FOUNDATION TRUST   (United Kingdom)

^d CAPITAL MEDICAL UNIVERSITY   (China)

Author keywords

AAV5; Gene therapy; OPN4; PMA; SH SY5Y; TPA

Indexed keywords

GREEN FLUORESCENT PROTEIN; PHORBOL 13 ACETATE 12 MYRISTATE; RECOMBINANT ADENO ASSOCIATED VIRUS TYPE 2; RETINOIC ACID; UNCLASSIFIED DRUG; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ADENO ASSOCIATED VIRUS TYPE 2; ARTICLE; CELL DIFFERENTIATION; CELL POPULATION; DRUG MECHANISM; HUMAN; HUMAN CELL; IN VITRO STUDY; INTRACELLULAR SIGNALING; NEUROBLASTOMA CELL; PRIORITY JOURNAL; PROTEIN EXPRESSION; SIGNAL TRANSDUCTION;

CARCINOGENS; CELL CYCLE PROTEINS; CELL DIFFERENTIATION; CELL DIVISION; DEPENDOVIRUS; DNA-BINDING PROTEINS; GENE EXPRESSION REGULATION; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; HEK293 CELLS; HUMANS; PROTEIN-SERINE-THREONINE KINASES; REAL-TIME POLYMERASE CHAIN REACTION; RECEPTORS, PLATELET-DERIVED GROWTH FACTOR; RETINAL NEOPLASMS; RETINOBLASTOMA; RNA, MESSENGER; TACROLIMUS BINDING PROTEINS; TETRADECANOYLPHORBOL ACETATE; TRANSDUCTION, GENETIC; TRETINOIN; TUMOR CELLS, CULTURED; TUMOR SUPPRESSOR PROTEINS;

EID: 84859491221     PISSN: 00144835     EISSN: 10960007     Source Type: Journal    
DOI: 10.1016/j.exer.2011.12.009     Document Type: Article

References (38)

1. Alexander J.J., Umino Y., Everhart D., Chang B., Min S.H., Li Q., Timmers A.M., Hawes N.L., Pang J.J., Barlow R.B., Hauswirth W.W. Restoration of cone vision in a mouse model of achromatopsia. Nat. Med. 2007, 13:685-687.
2. Biedler J.L., Roffler-Tarlov S., Schachner M., Freedman L.S. Multiple neurotransmitter synthesis by human neuroblastoma cell lines and clones. Cancer Res. 1978, 38:3751-3757.
3. Boye S.E., Boye S.L., Pang J., Ryals R., Everhart D., Umino Y., Neeley A.W., Besharse J., Barlow R., Hauswirth W.W. Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse. PLoS One 2010, 5:e11306.
4. Cataldi M.P., McCarty D.M. Differential effects of DNA double-strand break repair pathways on single-strand and self-complementary adeno-associated virus vector genomes. J. Virol. 2010, 84:8673-8682.
5. Cideciyan A.V. Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy. Prog. Retin. Eye Res. 2010, 29:398-427.
6. Di Pasquale G., Davidson B.L., Stein C.S., Martins I., Scudiero D., Monks A., Chiorini J.A. Identification of PDGFR as a receptor for AAV-5 transduction. Nat. Med. 2003, 9:1306-1312.
7. Donello J.E., Loeb J.E., Hope T.J. Woodchuck hepatitis virus contains a tripartite posttranscriptional regulatory element. J. Virol. 1998, 72:5085-5092.
8. Edwards I.J., Xu H., Obunike J.C., Goldberg I.J., Wagner W.D. Differentiated macrophages synthesize a heparan sulfate proteoglycan and an oversulfated chondroitin sulfate proteoglycan that bind lipoprotein lipase. Arterioscler Thromb. Vasc. Biol. 1995, 15:400-409.
9. Ferrari F.K., Samulski T., Shenk T., Samulski R.J. Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors. J. Virol. 1996, 70:3227-3234.
10. Flajolet M., Tiollais P., Buendia M.A., Fourel G. Woodchuck hepatitis virus enhancer I and enhancer II are both involved in N-myc2 activation in woodchuck liver tumors. J. Virol. 1998, 72:6175-6180.
11. Garrity-Moses M.E., Teng Q., Liu J., Tanase D., Boulis N.M. Neuroprotective adeno-associated virus Bcl-xL gene transfer in models of motor neuron disease. Muscle Nerve 2005, 32:734-744.
12. Hansen J., Qing K., Srivastava A. Adeno-associated virus type 2-mediated gene transfer: altered endocytic processing enhances transduction efficiency in murine fibroblasts. J. Virol. 2001, 75:4080-4090.
13. Hermanns J., Schulze A., Jansen-Db1urr P., Kleinschmidt J.A., Schmidt R., zur Hausen H. Infection of primary cells by adeno-associated virus type 2 results in a modulation of cell cycle-regulating proteins. J. Virol. 1997, 71:6020-6027.
14. Howard D.B., Powers K., Wang Y., Harvey B.K. Tropism and toxicity of adeno-associated viral vector serotypes 1, 2, 5, 6, 7, 8, and 9 in rat neurons and glia in vitro. Virology 2008, 372:24-34.
15. Kaplitt M.G., Feigin A., Tang C., Fitzsimons H.L., Mattis P., Lawlor P.A., Bland R.J., Young D., Strybing K., Eidelberg D., During M.J. Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial. Lancet 2007, 369:2097-2105.
16. Khani S.C., Pawlyk B.S., Bulgakov O.V., Kasperek E., Young J.E., Adamian M., Sun X., Smith A.J., Ali R.R., Li T. AAV-mediated expression targeting of rod and cone photoreceptors with a human rhodopsin kinase promoter. Invest. Ophthalmol. Vis. Sci. 2007, 48:3954-3961.
17. Kingsman S.M., Mitrophanous K., Olsen J.C. Potential oncogene activity of the woodchuck hepatitis post-transcriptional regulatory element (WPRE). Gene Ther. 2005, 12:3-4.
18. Lee J.H., Paull T.T. ATM activation by DNA double-strand breaks through the Mre11-Rad50-Nbs1 complex. Science 2005, 308:551-554.
19. Li M., Jayandharan G.R., Li B., Ling C., Ma W., Srivastava A., Zhong L. High-efficiency transduction of fibroblasts and mesenchymal stem cells by tyrosine-mutant AAV2 vectors for their potential use in cellular therapy. Hum. Gene Ther. 2010, 21:1527-1543.
20. Mancuso K., Hauswirth W.W., Li Q., Connor T.B., Kuchenbecker J.A., Mauck M.C., Neitz J., Neitz M. Gene therapy for red-green colour blindness in adult primates. Nature 2009, 461:784-787.
21. Nitti M., Furfaro A.L., Cevasco C., Traverso N., Marinari U.M., Pronzato M.A., Domenicotti C. PKC delta and NADPH oxidase in retinoic acid-induced neuroblastoma cell differentiation. Cell Signal. 2010, 22:828-835.
22. Nomoto T., Okada T., Shimazaki K., Mizukami H., Matsushita T., Hanazono Y., Kume A., Katsura K., Katayama Y., Ozawa K. Distinct patterns of gene transfer to gerbil hippocampus with recombinant adeno-associated virus type 2 and 5. Neurosci. Lett. 2003, 340:153-157.
23. Pahlman S., Mamaeva S., Meyerson G., Mattsson M.E., Bjelfman C., Ortoft E., Hammerling U. Human neuroblastoma cells in culture: a model for neuronal cell differentiation and function. Acta Physiol. Scand. Suppl. 1990, 592:25-37.
24. Pahlman S., Ruusala A.I., Abrahamsson L., Mattsson M.E., Esscher T. Retinoic acid-induced differentiation of cultured human neuroblastoma cells: a comparison with phorbolester-induced differentiation. Cell Differ. 1984, 14:135-144.
25. Pang J.J., Alexander J., Lei B., Deng W., Zhang K., Li Q., Chang B., Hauswirth W.W. Achromatopsia as a potential candidate for gene therapy. Adv. Exp. Med. Biol. 2010, 664:639-646.
26. Peirson S.N., Butler J.N., Foster R.G. Experimental validation of novel and conventional approaches to quantitative real-time PCR data analysis. Nucleic Acids Res. 2003, 31:e73.
27. Samulski R.J., Chang L.S., Shenk T. A recombinant plasmid from which an infectious adeno-associated virus genome can be excised in vitro and its use to study viral replication. J. Virol. 1987, 61:3096-3101.
28. Sanlioglu S., Benson P., Engelhardt J.F. Loss of ATM function enhances recombinant adeno-associated virus transduction and integration through pathways similar to UV irradiation. Virology 2000, 268:68-78.
29. Teramoto S., Bartlett J.S., McCarty D., Xiao X., Samulski R.J., Boucher R.C. Factors influencing adeno-associated virus-mediated gene transfer to human cystic fibrosis airway epithelial cells: comparison with adenovirus vectors. J. Virol. 1998, 72:8904-8912.
30. Teschendorf C., Emons B., Muzyczka N., Graeven U., Schmiegel W. Efficacy of recombinant adeno-associated viral vectors serotypes 1, 2, and 5 for the transduction of pancreatic and colon carcinoma cells. Anticancer Res. 2010, 30:1931-1935.
31. Truman J.P., Rotenberg S.A., Kang J.H., Lerman G., Fuks Z., Kolesnick R., Marquez V.E., Haimovitz-Friedman A. PKCalpha activation downregulates ATM and radio-sensitizes androgen-sensitive human prostate cancer cells in vitro and in vivo. Cancer Biol. Ther. 2009, 8:54-63.
32. Vandenberghe L.H., Bell P., Maguire A.M., Cearley C.N., Xiao R., Calcedo R., Wang L., Castle M.J., Maguire A.C., Grant R., Wolfe J.H., Wilson J.M., Bennett J. Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkey. Sci. Transl Med. 2011, 3:88ra54.
33. Vandesompele J., De Preter K., Pattyn F., Poppe B., Van Roy N., De Paepe A., Speleman F. Accurate normalization of real-time quantitative RT-PCR data by geometric averaging of multiple internal control genes. Genome Biol. 2002, 3. RESEARCH0034.
34. Wang J., Xie J., Lu H., Chen L., Hauck B., Samulski R.J., Xiao W. Existence of transient functional double-stranded DNA intermediates during recombinant AAV transduction. Proc. Natl. Acad. Sci. U S A 2007, 104:13104-13109.
35. Zanta-Boussif M.A., Charrier S., Brice-Ouzet A., Martin S., Opolon P., Thrasher A.J., Hope T.J., Galy A. Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: application to the gene therapy of WAS. Gene Ther. 2009, 16:605-619.
36. Zentilin L., Marcello A., Giacca M. Involvement of cellular double-stranded DNA break binding proteins in processing of the recombinant adeno-associated virus genome. J. Virol. 2001, 75:12279-12287.
37. Zhao W., Zhong L., Wu J., Chen L., Qing K., Weigel-Kelley K.A., Larsen S.H., Shou W., Warrington K.H., Srivastava A. Role of cellular FKBP52 protein in intracellular trafficking of recombinant adeno-associated virus 2 vectors. Virology 2006, 353:283-293.
38. Zhong L., Li W., Yang Z., Chen L., Li Y., Qing K., Weigel-Kelley K.A., Yoder M.C., Shou W., Srivastava A. Improved transduction of primary murine hepatocytes by recombinant adeno-associated virus 2 vectors in vivo. Gene Ther. 2004, 11:1165-1169.