Synthesis of a modified gene encoding human ornithine transcarbamylase for expression in mammalian mitochondrial and universal translation systems: A novel approach towards correction of a genetic defect

Gene

Volumn 169, Issue 2, 1996, Pages 251-255

  Wheeler, Vanessa C ^a   Prodromou, Chrisostomos ^b   Pearl, Laurence H ^b   Williamson, Robert ^a   Coutelle, Charles ^a  

^a IMPERIAL COLLEGE LONDON   (United Kingdom)

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

Chaperone; Gene transfer; Genetic code; Organelle; OTC deficiency; Recursive PCR; Urea cycle

Indexed keywords

MITOCHONDRIAL DNA; ORNITHINE CARBAMOYLTRANSFERASE; SYNTHETIC DNA;

ARTICLE; DNA FLANKING REGION; GENE EXPRESSION; GENE TRANSFER; HUMAN; MAMMAL; NONHUMAN; NUCLEOTIDE SEQUENCE; POLYACRYLAMIDE GEL ELECTROPHORESIS; PRIORITY JOURNAL; RNA TRANSLATION;

AMINO ACID SEQUENCE; ANIMALS; BASE SEQUENCE; CELL LINE; GENE EXPRESSION; GENE TRANSFER TECHNIQUES; GENETIC DISEASES, INBORN; HUMANS; MAMMALS; MITOCHONDRIA; MOLECULAR SEQUENCE DATA; ORNITHINE CARBAMOYLTRANSFERASE; PROTEIN BIOSYNTHESIS;

EID: 0030002009     PISSN: 03781119     EISSN: None     Source Type: Journal    
DOI: 10.1016/0378-1119(95)00812-8     Document Type: Article

References (25)

1. Bambot, S.B. and Russell, A.J.: Efficient total gene synthesis of 1.35 kb hybrid a-lytic protease gene using the polymerase chain reaction. PCR Methods Appl. 2 (1993) 266-271.
2. Cariello, N.F., Swenberg, J.A. and Skopeck, T.R.: Fidelity of Thermococcus litoralis DNA polymerase (Vent ™) in PCR determined by denaturing gradient gel electrophoresis. Nucleic Acids Res. 19 (1991) 4193-4198.
3. Cavard, C., Grimber, G., Duboi, N., Chasse, J.-F., Benoun, M., Minet-Thuriaux, M., Kamoun, P. and Briand, P.: Correction of mouse ornithine transcarbamylase-deficiency by gene transfer into the germ line. Nucleic Acids Res. 16 (1988) 2099-2110.
4. Clayton, D. A.: Replication of animal mitochondrial DNA. Cell 28 (1982) 693-705.
5. Clayton, D.A.: Transcription of the mammalian mitochondrial genome. Annu. Rev. Biochem. 53 (1984) 573-594.
6. Crabeel, M., Charlier, D., Cunin, R. and Glansdorff, N.: Cloning and endonuclease restriction analysis of argF and of the control region of the argECBH bipolar operon in Escherichia coli. Gene 5 (1978) 207-231.
7. Doolitle, D.P., Hulpert, L.L. and Cordy, C.A.: A new allele of the sparse fur gene in the mouse. J. Hered. 65 (1974) 194-195.
8. Grompe, M., Jones, S.N., Loulseged, H. and Caskey, C.T.: Retroviral-mediated gene transfer of human ornithine transcarbamylase into primary hepatocytes of spf and spf-ash mice. Hum. Gene Ther. 3 (1992) 35-44.
9. Jones, S.N., Grompe, M., Munir, M.I., Veres, G., Craigen, W.J. and Caskey, C.T.: Ectopic correction of ornithine transcarbamylase deficiency in sparse fur mice. J. Biol. Chem. 265 (1990) 14684-14690.
10. Ling, L.L., Keohavong, P., Dias, C. and Thilly, W.G.: Optimization of the polymerase chain reaction with regard to fidelity: modified T7, Taq, and Vent DNA polymerases. PCR Methods Appl. 1 (1991) 63-69.
11. Majumder, K.: Ligation-free gene synthesis by PCR: synthesis and mutagenesis at multiple loci of a chimeric gene encoding OmpA signal peptide and hirudin. Gene 110 (1992) 89-94.
12. Mattila, P., Korpela, J., Tenkanen, T. and Pitkänen, K.: Fidelity of DNA synthesis by the Thermococcus litoralis DNA polymerase-an extremely heat stable enzyme with proofreading activity. Nucleic Acids Res. 19 (1991) 4967-4973.
13. McLennan, N.F., Girshovich, A.S., Lissin, N.M., Charters, Y. and Masters, M.: The strongly conserved C terminus glycine-methionine motif of the Escherichia coli GroEL chaperonin is dispensable. Mol. Microbiol. 7 (1993) 49-58.
14. Morsy, M.A., Alford, E.L., Bett, A., Graham, F.L. and Caskey, C.T.: Efficient adenoviral-mediated ornithine transcarbamylase expression in deficient mouse and human hepatocytes. J. Clin. Invest. 92 (1993) 1580-1586.
15. Murakami, K., Tokunaga, F., Iwanaga, S. and Mori, M.: Presequence does not prevent folding of a purified mitochondrial precursor protein and is essential for association with a reticulocyte cytosolic factor(s). J. Biochem. 108 (1990) 207-214.
16. Prescott, L.M. and Jones, M.E.: Modified methods for the determination of carbamyl aspartate. Anal. Biochem. 32 (1969) 408-419.
17. Prodromou, C. and Pearl, L.H.: Recursive PCR: a novel technique for total gene synthesis. Protein Eng. 5 (1992) 827-829.
18. Rosenberg, L.E., Fenton, W.A., Horwich, A.L., Kalousek, F. and Kraus, J.P.: Targetting of nuclear-encoded proteins to the mitochondrial matrix: implications for human genetic defects. Ann. N.Y. Acad. Sci. 488 (1987) 99-108.
19. Saiki, R.K., Gelfand, D.H., Stoffel, S., Scharf, S.J., Higuchi, R., Horn, G.T., Mullis, K.B. and Erlich, H.A.: Primer-directed enzymatic amplification of DNA with a thermostable DNA polymerase. Science 239 (1988) 487-491.
20. Sandhu, G.S., Aleff, R.A. and Kline, B.C.: Dual asymmetric PCR: one-step construction of synthetic genes. Biotechniques 12 (1992) 14-16.
21. Stratford-Perricaudet, L.D., Levrero, M., Chasse, J.F., Perricaudet, M. and Briand, P.: Evaluation of the transfer and expression in mice of an enzyme encoding gene using a human adenovirus vector. Hum. Gene Ther. 1 (1990) 241-256.
22. Veres, G., Gibbs, R.A., Scherer, S.E. and Caskey, C.T.: The molecular basis of the sparse fur mouse mutation. Science 237 (1987) 415-417.
23. Walser, M.: Urea cycle disorders and other hereditory hyperammonemic syndromes. In: Stanbury, J.B., Wyngaarden, J.B., Fredrickson, D.S., Goldstein, J.L. and Brown, M.S. (Eds.), The Metabolic Basis of Inherited Disease. McGraw-Hill, New York, NY, 1983, pp. 402-438.
24. Ye, Q.-Z., Johnson, L.L. and Baragi, V.: Gene synthesis and expression in E. coli for Pump, a human matrix metalloproteinase. Biochem. Biophys. Res. Commun. 186 (1992) 143-149.
25. Zheng, X., Rosenberg, L.E., Kolousek, F. and Fenton, W.A.: GroEL, GroES, and ATP-dependent folding and spontaneous assembly of ornithine transcarbamylase. J. Biol. Chem. 268 (1993) 7489-7493.