Rescue of splicing-mediated intron loss maximizes expression in lentiviral vectors containing the human ubiquitin C promoter

Nucleic Acids Research

Volumn 43, Issue 1, 2015, Pages 682-690

  Cooper, Aaron R ^a   Lill, Georgia R ^a   Gschweng, Eric H ^a   Kohn, Donald B ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

EEF1A1 PROTEIN, HUMAN; ELONGATION FACTOR 1; UBIQUITIN C;

ENHANCER REGION; EXON; GENE EXPRESSION; GENE VECTOR; GENETICS; HEK293 CELL LINE; HUMAN; INTRON; K562 CELL LINE; LENTIVIRINAE; PROMOTER REGION; RNA SPLICING;

ENHANCER ELEMENTS, GENETIC; EXONS; GENE EXPRESSION; GENETIC VECTORS; HEK293 CELLS; HUMANS; INTRONS; K562 CELLS; LENTIVIRUS; PEPTIDE ELONGATION FACTOR 1; PROMOTER REGIONS, GENETIC; RNA SPLICING; UBIQUITIN C;

EID: 84943278442     PISSN: 03051048     EISSN: 13624962     Source Type: Journal    
DOI: 10.1093/nar/gku1312     Document Type: Article

References (29)

1. Zufferey,R., Dull,T., Mandel,R.J., Bukovsky,A., Quiroz,D., Naldini,L. and Trono,D. (1998) Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. J. Virol., 72, 9873-9880.
2. Schambach,A., Bohne,J., Chandra,S.,Will,E., Margison,G.P., Williams,D.A. and Baum,C. (2006) Equal potency of gammaretroviral and lentiviral SIN vectors for expression of O6-methylguanine-DNA methyltransferase in hematopoietic cells. Mol. Ther., 13, 391-400.
3. Dull,T., Zufferey,R., Kelly,M., Mandel,R.J., Nguyen,M., Trono,D. and Naldini,L. (1998) A third-generation lentivirus vector with a conditional packaging system. J. Virol., 72, 8463-8471.
4. Ramezani,A., Hawley,T.S. and Hawley,R.G. (2000) Lentiviral vectors for enhanced gene expression in human hematopoietic cells. Mol. Ther., 2, 458-469.
5. Zaiss,A.-K., Son,S. and Chang,L.-J. (2002) RNA 3readthrough of oncoretrovirus and lentivirus: implications for vector safety and efficacy. J. Virol., 76, 7209-7219.
6. Logan,A.C., Lutzko,C. and Kohn,D.B. (2002) Advances in lentiviral vector design for gene-modification of hematopoietic stem cells. Curr. Opin. Biotechnol., 13, 429-436.
7. Bianchi,M., Crinelli,R., Giacomini,E., Carloni,E. and Magnani,M. (2009) A potent enhancer element in the 5-UTR intron is crucial for transcriptional regulation of the human ubiquitin C gene. Gene, 448, 88-101.
8. Lois,C., Hong,E.J., Pease,S., Brown,E.J. and Baltimore,D. (2002) Germline transmission and tissue-specific expression of transgenes delivered by lentiviral vectors. Science, 295, 868-872.
9. Kamata,M., Liang,M., Liu,S., Nagaoka,Y. and Chen,I.S.Y. (2010) Live cell monitoring of hiPSC generation and differentiation using differential expression of endogenous microRNAs. PLoS ONE, 5, e11834.
10. Joglekar,A.V., Hollis,R.P., Kuftinec,G., Senadheera,S., Chan,R. and Kohn,D.B. (2013) Integrase-defective lentiviral vectors as a delivery platform for targeted modification of adenosine deaminase locus. Mol. Ther., 21, 1705-1717.
11. Zufferey,R., Nagy,D., Mandel,R.J., Naldini,L. and Trono,D. (1997) Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo. Nat. Biotechnol., 15, 871-875.
12. Hanawa,H., Kelly,P.F., Nathwani,A.C., Persons,D.A., Vandergriff,J.A., Hargrove,P., Vanin,E.F. and Nienhuis,A.W. (2002) Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transduce primitive hematopoietic cells from human blood. Mol. Ther., 5, 242-251.
13. Maetzig,T., Galla,M., Brugman,M.H., Loew,R., Baum,C. and Schambach,A. (2010) Mechanisms controlling titer and expression of bidirectional lentiviral and gammaretroviral vectors. Gene Ther., 17, 400-411.
14. Tsien,R.Y. (1998) The green fluorescent protein. Annu. Rev. Biochem., 67, 509-544.
15. Bianchi,M., Crinelli,R., Giacomini,E., Carloni,E., Radici,L. and Magnani,M. (2013) Yin Yang 1 intronic binding sequences and splicing elicit intron-mediated enhancement of ubiquitin C gene expression. PLoS ONE, 8, e65932.
16. Amendola,M., Venneri,M.A., Biffi,A., Vigna,E. and Naldini,L. (2005) Coordinate dual-gene transgenesis by lentiviral vectors carrying synthetic bidirectional promoters. Nat. Biotechnol., 23, 108-116.
17. Cartier,N., Hacein-Bey-Abina,S., Bartholomae,C.C., Veres,G., Schmidt,M., Kutschera,I., Vidaud,M., Abel,U., Dal Cortivo,L., Caccavelli,L. et al. (2009) Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science, 326, 818-823.
18. Cavazzana-Calvo,M., Payen,E., Negre,O., Wang,G., Hehir,K., Fusil,F., Down,J., Denaro,M., Brady,T., Westerman,K. et al. (2010) Transfusion independence and HMGA2 activation after gene therapy of human-thalassaemia. Nature, 467, 318-322.
19. Aiuti,A., Biasco,L., Scaramuzza,S., Ferrua,F., Cicalese,M.P., Baricordi,C., Dionisio,F., Calabria,A., Giannelli,S., Castiello,M.C. et al. (2013) Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science, 341, 1233151.
20. Biffi,A., Montini,E., Lorioli,L., Cesani,M., Fumagalli,F., Plati,T., Baldoli,C., Martino,S., Calabria,A., Canale,S. et al. (2013) Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science, 341, 1233158.
21. Romero,Z., Urbinati,F., Geiger,S., Cooper,A.R., Wherley,J., Kaufman,M.L., Hollis,R.P., de Assin,R.R., Senadheera,S., Sahagian,A. et al. (2013)-globin gene transfer to human bone marrow for sickle cell disease. J. Clin. Invest., 123, 3317-3330.
22. Carbonaro,D.A., Zhang,L., Jin,X., Montiel-Equihua,C., Geiger,S., Carmo,M., Cooper,A., Fairbanks,L., Kaufman,M.L., Sebire,N.J. et al. (2014) Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency. Mol. Ther., 22, 607-622.
23. Morcos,P.A. (2007) Achieving targeted and quantifiable alteration of mRNA splicing with Morpholino oligos. Biochem. Biophys. Res. Commun., 358, 521-527.
24. Hacein-Bey Abina,S., von Kalle,C., Schmidt,M., McCormack,M.P., Wulffraat,N., Leboulch,P., Lim,A., Osborne,C.S., Pawliuk,R., Morillon,E. et al. (2003) LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science, 302, 415-419.
25. Hacein-Bey-Abina,S., Garrigue,A., Wang,G.P., Soulier,J., Lim,A., Morillon,E., Clappier,E., Caccavelli,L., Delabesse,E., Beldjord,K. et al. (2008) Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J. Clin. Invest., 118, 3132-3142.
26. Howe,S.J., Mansour,M.R., Schwarzwaelder,K., Bartholomae,C., Hubank,M., Kempski,H., Brugman,M.H., Pike-Overzet,K., Chatters,S.J., de Ridder,D. et al. (2008) Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J. Clin. Invest., 118, 3143-3150.
27. Stein,S., Ott,M.G., Schultze-Strasser,S., Jauch,A., Burwinkel,B., Kinner,A., Schmidt,M., Krämer,A., Schwäble,J., Glimm,H. et al. (2010) Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. Nat. Med., 16, 198-204.
28. Zheng,Z.-M. (2004) Regulation of alternative RNA splicing by exon definition and exon sequences in viral and mammalian gene expression. J. Biomed. Sci., 11, 278-294.
29. Pandya-Jones,A., Bhatt,D.M., Lin,C.-H., Tong,A.-J., Smale,S.T. and Black,D.L. (2013) Splicing kinetics and transcript release from the chromatin compartment limit the rate of Lipid A-induced gene expression. RNA, 19, 811-827.