Strong muscle-specific regulatory cassettes based on multiple copies of the human slow troponin I gene upstream enhancer

Human Gene Therapy

Volumn 21, Issue 1, 2010, Pages 127-134

  Blain, Marilyne ^a,b   Zeng, Yue ^a,c   Bendjelloul, Mehdi ^a,c   Hallauer, Patricia L ^c   Kumar, Angela ^c   Hastings, Kenneth E M ^c   Karpati, George ^c   Massie, Bernard ^a,b   Gilbert, Rénald ^a,c  

^a NATIONAL RESEARCH COUNCIL CANADA   (Canada)

^b UNIVERSITÉ DE MONTRÉAL   (Canada)

^c MCGILL UNIVERSITY   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

BETA ACTIN; BETA GALACTOSIDASE; PLASMID DNA; TROPONIN I;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; CYTOMEGALOVIRUS; ELECTROPORATION; ENHANCER REGION; FAST MUSCLE FIBER; GENE CASSETTE; GENE CONSTRUCT; GENE DELETION; GENE EXPRESSION; GENETIC TRANSCRIPTION; HUMAN; HUMAN CELL; HYBRID; HYDRODYNAMICS BASED TRANSFECTION; MOUSE; MYOTUBE; NONHUMAN; NUCLEOTIDE SEQUENCE; PROMOTER REGION; SLOW MUSCLE FIBER; SOLEUS MUSCLE; TIBIALIS ANTERIOR MUSCLE; TISSUE SPECIFICITY;

ANIMALS; BASE SEQUENCE; BETA-GALACTOSIDASE; CELLS, CULTURED; ENHANCER ELEMENTS, GENETIC; GENE DOSAGE; GENE EXPRESSION REGULATION; GENES, REPORTER; GENETIC ENGINEERING; HUMANS; MICE; MUSCLE FIBERS, FAST-TWITCH; MUSCLE FIBERS, SLOW-TWITCH; MUSCLE, SKELETAL; ORGAN SPECIFICITY; PLASMIDS; TROPONIN I;

CYTOMEGALOVIRUS; MUS MUSCULUS;

EID: 74949100722     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2008.208     Document Type: Article

References (27)

1. Calvo, S., Vullhorst, D., Venepally, P., Cheng, J., Karavanova, I., and Buonanno, A. (2001). Molecular dissection of DNA sequences and factors involved in slow muscle-specific transcription. Mol. Cell Biol. 21, 8490-8503.
2. Cordier, L., Gao, G.P., Hack, A.A., McNally, E.M., Wilson, J.M., Chirmule, N., and Sweeney, H.L. (2001). Muscle-specific promoters may be necessary for adeno-associated virus-mediated gene transfer in the treatment of muscular dystrophies. Hum. Gene Ther. 12, 205-215.
3. Corin, S.J., Levitt, L.K., O'Mahoney, J.V., Joya, J.E., Hardeman, E.C., and Wade, R. (1995). Delineation of a slow-twitch-myofiber-specific transcriptional element by using in vivo somatic gene transfer. Proc. Natl. Acad. Sci. U. S. A. 92, 6185-6189.
4. Gaillet, B., Gilbert, R., Amziani, R., Guilbault, C., Gadoury, C., Caron, A.W., Mullick, A., Garnier, A., and Massie, B. (2007). High-level recombinant protein production in CHO cells using an adenoviral vector and the cumate gene-switch. Bio-technol. Prog. 23, 200-209.
5. Gilbert, R., Liu, A., Petrof, B., Nalbantoglu, J., and Karpati, G. (2002). Improved performance of a fully gutted adenovirus vector containing two full-length dystrophin cDNAs regulated by a strong promoter. Mol. Ther. 6, 501-509.
6. Hallauer, P.L., Bradshaw, H.L., and Hastings, K.E. (1993). Complex fiber-type-specific expression of fast skeletal muscle troponin I gene constructs in transgenic mice. Development 119, 691-701.
7. Hallauer, P.L., and Hastings, K.E. (2002). TnIfast IRE enhancer: Multistep developmental regulation during skeletal muscle fiber type differentiation. Dev. Dyn. 224, 422-431.
8. Hartigan-O'Connor, D., Kirk, C.J., Crawford, R., Mule, J.J., and Chamberlain, J.S. (2001). Immune evasion by muscle-specific gene expression in dystrophic muscle. Mol. Ther. 4, 525-533.
9. Hauser, M.A., Robinson, A., Hartigan-O'Connor, D., Williams-Gregory, D.A., Buskin, J.N., Apone, S., Kirk, C.J., Hardy, S., Hauschka, S.D., and Chamberlain, J.S. (2000). Analysis of muscle creatine kinase regulatory elements in recombinant adenoviral vectors. Mol. Ther. 2, 16-25.
10. Ishii, A., Hagiwara, Y., Saito, Y., Yamamoto, K., Yuasa, K., Sato, Y., Arahata, K., Shoji, S., Nonaka, I., Saito, I., Nabeshima, Y., and Takeda, S. (1999). Effective adenovirus-mediated gene expression in adult murine skeletal muscle. Muscle Nerve 22, 592-599.
11. Issa, L.L., Palmer, S.J., Guven, K.L., Santucci, N., Hodgson, V.R., Popovic, K., Joya, J.E., and Hardeman, E.C. (2006). MusTRD can regulate postnatal fiber-specific expression. Dev. Biol. 293, 104-115.
12. Larochelle, N., Lochmuller, H., Zhao, J., Jani, A., Hallauer, P., Hastings, K.E., Massie, B., Prescott, S., Petrof, B.J., Karpati, G., and Nalbantoglu, J. (1997). Efficient muscle-specific transgene expression after adenovirus-mediated gene transfer in mice using a 1.35kb muscle creatine kinase promoter=enhancer. Gene Ther. 4, 465-472.
13. Larochelle, N., Oualikene, W., Dunant, P., Massie, B., Karpati, G., Nalbantoglu, J., and Lochmuller, H. (2002). The short MCK1350 promoter=enhancer allows for sufficient dystrophin expression in skeletal muscles of mdx mice. Biochem. Biophys. Res. Commun. 292, 626-631.
14. Levitt, L.K., O'Mahoney, J.V., Brennan, K.J., Joya, J.E., Zhu, L., Wade, R.P., and Hardeman, E.C. (1995). The human troponin I slow promoter directs slow fiber-specific expression in trans-genic mice. DNA Cell Biol. 14, 599-607.
15. Li, X., Eastman, E.M., Schwartz, R.J., and Draghia-Akli, R. (1999). Synthetic muscle promoters: Activities exceeding naturally occurring regulatory sequences. Nat. Biotechnol. 17, 241-245.
16. Liu, F., Song, Y., and Liu, D. (1999). Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA. Gene Ther. 6, 1258-1266.
17. Lu, Q.L., Bou-Gharios, G., and Partridge, T.A. (2003). Non-viral gene delivery in skeletal muscle: A protein factory. Gene Ther. 10, 131-142.
18. Molnar, M.J., Gilbert, R., Lu, Y., Liu, A.B., Guo, A., Larochelle, N., Orlopp, K., Lochmuller, H., Petrof, B.J., Nalbantoglu, J., and Karpati, G. (2004). Factors influencing the efficacy, longevity, and safety of electroporation-assisted plasmid-based gene transfer into mouse muscles. Mol. Ther. 10, 447-455.
19. Nakayama, M., Stauffer, J., Cheng, J., Banerjee-Basu, S., Wawrousek, E., and Buonanno, A. (1996). Common core sequences are found in skeletal muscle slow-and fast-fiber-type-specific regulatory elements. Mol. Cell Biol. 16, 2408-2417.
20. Niwa, H., Yamamura, K., and Miyazaki, J. (1991). Efficient selection for high-expression transfectants with a novel eu-karyotic vector. Gene 108, 193-199.
21. Odom, G.L., Gregorevic, P., and Chamberlain, J.S. (2007). Viral-mediated gene therapy for the muscular dystrophies: Successes, limitations and recent advances. Biochim. Biophys. Acta 1772, 243-262.
22. Polly, P., Haddadi, L.M., Issa, L.L., Subramaniam, N., Palmer, S.J., Tay, E.S., and Hardeman, E.C. (2003). hMusTRD1alpha1 represses MEF2 activation of the troponin I slow enhancer. J. Biol. Chem. 278, 36603-36610.
23. Ratanamart, J., and Shaw, J.A. (2006). Plasmid-mediated muscle-targeted gene therapy for circulating therapeutic protein replacement: A tale of the tortoise and the hare? Curr. Gene Ther. 6, 93-110.
24. Salva, M.Z., Himeda, C.L., Tai, P.W., Nishiuchi, E., Gregorevic, P., Allen, J.M., Finn, E.E., Nguyen, Q.G., Blankinship, M.J., Meuse, L., Chamberlain, J.S., and Hauschka, S.D. (2007). Design of tissue-specific regulatory cassettes for high-level rAAV-mediated expression in skeletal and cardiac muscle. Mol. Ther. 15, 320-329.
25. Schiaffino, S., Gorza, L., Sartore, S., Saggin, L., Ausoni, S., Via-nello, M., Gundersen, K., and Lomo, T. (1989). Three myosin heavy chain isoforms in type 2 skeletal muscle fibres. J. Muscle Res. Cell Motil. 10, 197-205.
26. Wang, B., Li, J., Fu, F.H., Chen, C., Zhu, X., Zhou, L., Jiang, X., and Xiao, X. (2008). Construction and analysis of compact muscle-specific promoters for AAV vectors. Gene Ther. 15, 1489-1499.
27. Zhang, G., Budker, V., and Wolff, J.A. (1999). High levels of foreign gene expression in hepatocytes after tail vein injections of naked plasmid DNA. Hum. Gene Ther. 10, 1735-1737.