Synthesis and processing of genetically modified human proinsulin by rat myoblast primary cultures

Human Gene Therapy

Volumn 7, Issue 1, 1996, Pages 71-78

  Simonson, Gregg D ^a   Groskreutz, Debrya J ^a,b   Gorman, Cornelia M ^a,c   MacDonald, Michael J ^a,d  

^a UNIVERSITY OF WISCONSIN MADISON   (United States)

^b PROMEGA CORPORATION   (United States)

^c Megabios Corporation   (United States)

^d UNIVERSITY OF WISCONSIN SCHOOL OF MEDICINE AND PUBLIC HEALTH   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ASPARTIC ACID; BETA GALACTOSIDASE; COMPLEMENTARY DNA; HISTIDINE; PROINSULIN;

ADIPOCYTE; ANIMAL CELL; ARTICLE; CONTROLLED STUDY; CULTURE MEDIUM; CYTOMEGALOVIRUS; DNA CLEAVAGE; DNA TRANSFECTION; DRUG STABILITY; EXPRESSION VECTOR; GENE THERAPY; GENETIC ENGINEERING; HORMONE SYNTHESIS; INSULIN DEPENDENT DIABETES MELLITUS; MOLECULAR CLONING; MYOBLAST; MYOTUBE; NEWBORN; NONHUMAN; POINT MUTATION; RAT;

EID: 0029922307     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.1996.7.1-71     Document Type: Article

References (42)

1. BLAU, H.M., DHAWAN, J., and PAVLATH, G.K. (1993). Myoblasts in pattern formation and gene therapy. Trends Genet. 9, 269-274.
2. BOSHART, M., WEBER, F., JAHN, G., DORSCH-HASLER, K., FLECKENSTEIN, B., and SCHAFFNER, W. (1985). A very strong enhancer is located upstream of an immediate early gene of human cytomegalovirus. Cell 41, 521-528.
3. BRANGE, J., OWENS, D.R., KING, S., and VOLUND, A. (1990). Monomeric insulins and their experimental and clinical implications. Diabetes Care 13, 923-954.
4. BREMS, D.N., BROWN, P.L., BRYANT, C., CHANCE, R.E., GREEN, L.K., LONG, H.B., MILLER, A.A., MILLICAN, R., SHIELDS, J.E., and FRANK, B.H. (1992). Improved insulin stability through amino acid substitution. Protein Eng. 5, 519-525.
5. 10 amino acid residue. Requirements of high biological activity insulin. Int. J. Protein Res. 23, 394-401.
6. CARROLL, R.J., HAMMER, R.E., CHAN, S.J., SWIFT, H.H., RUBENSTEIN, A.H., and STEINER, D.F. (1988). A mutant human proinsulin is secreted from islets of Langerhans in increased amounts via an unregulated pathway. Proc. Natl. Acad. Sci. USA 85, 8943-8947.
7. CHAN, S.J., SEINO, S., GRUPPUSO, P.A., SCHWARTZ, R., and STEINER, D.F. (1987). A mutation in the B chain coding region is associated with impaired proinsulin conversion in a family with hyperproinsulinemia. Proc. Natl. Acad. Sci. USA 84, 2194-2197.
8. DAI, Y., ROMAN, M., NAVIAUX, R.K., and VERMA, I.M. (1992). Gene therapy via primary myoblasts, long-term expression of factor IX protein following transplantation in vivo. Proc. Natl. Acad. Sci. USA 89, 10892-10895.
9. DAVIDSON, H.W., RHODES, C.J., and HUTTON, J.C. (1988). Intraorganellar calcium and pH control proinsulin cleavage in the pancreatic β cell via two site-specific endopeptidases. Nature 333, 93-96.
10. DAVIS, M.G., and HUANG, E.S. (1988). Transfer and expression of plasmids containing human cytomegalovirus immediate-early gene 1 promoter-enhancer sequences in eukaryotic and prokaryotic cells. Biotech. & Applied Biochem. 10, 6-12.
11. DHAWAN, J., PAN, L.C., PAVLATH, G.K., TRAVIS, M.A., LANCTOT, A.M., and BLAU, H.M. (1991). Systemic delivery of human growth hormone by injection of genetically engineered myoblasts. Science 254, 1509-1512.
12. B10]insulin in AtT20 cells. Mol. Endocrinol. 5, 319-326.
13. FOECKING, M.K., and HOFSTETTER, H. (1986). Powerful and versatile enhancer-promoter unit for mammalian expression vectors. Gene 45, 101-105.
14. FULLER, R.S., BRAKE, A.J., and THONER, J. (1989). Intracellular targeting and structural conservation of a prohormone-processing endoprotease. Science 246, 482-486.
15. GLIEMANN, J. (1965). Insulin-like activity of dilute human serum assayed by an isolated adipose cell method. Diabetes 14, 643-649.
16. GLIEMANN, J. (1967). Assay of insulin-like activity by the isolated fat cell method. Diabetologia 3, 382-388.
17. GROSKREUTZ, D.J., SLIWKOWSKI, M.X., and GORMAN, C.M. (1994). Genetically engineered proinsulin constitutively processed and secreted as mature, active insulin. J. Biol. Chem. 269, 6241-6245.
18. GROSS, D.J., HALBAN, P.A., KAHN, C.R., WEIR, G.C., and VILLA-KOMAROFF, L. (1989). Partial diversion of a mutant proinsulin (B10 aspartic acid) from the regulated to the constitutive secretory pathway in transfected AtT-20 cells. Proc. Natl. Acad. Sci. USA 86, 4107-4111.
19. HARLOW, E., and LANE, D. (1988). Antibodies: A Laboratory Manual. (Cold Spring Laboratory, Cold Spring Harbor, New York).
20. HUGHES, S., and BLAU, H. (1990). Migration of myoblasts across basal lamina during skeletal muscle development. Nature 345, 350-353.
21. KONIGSBERG, I.R. (1979) Skeletal myoblasts in culture. Methods Enzymol. 58, 511-527.
22. LYNCH, C.M., CLOWES, M.M., OSBORNE, W.R.A., CLOWES, A.W., and MILLER, A.D. (1992). Long-term expression of human adenosine deaminase in vascular smooth muscle cells of rats: A model for gene therapy. Proc. Natl. Acad. Sci. USA 89, 1138-1142.
23. MACDONALD, M.J., and GREWE, B.K. (1981). Inhibition of phosphoenolpyruvate carboxykinase, glyceroneogenesis and fatty acid synthesis in rat adipose tissue by quinolinate and 3-mercaptopicolinate. Biochim. Biophys. Acta 663, 302-313.
24. MIZUNO, K., NAKAMURA, T., OSHIMA, T., TANAKA, S., and MATSUO, H. (1988). Yeast KEX2 genes encodes an endopeptidase homologous to subtilisin-like proteases. Biochem. Biophys. Res. Commun. 156, 246-254.
25. PARTRIDGE, T.A. (1991). Invited review. Myoblast transfer: A possible therapy for inherited myopathies. Muscle & Nerve 14, 197-212.
26. PARTRIDGE, T.A., MORGAN, J.E., COULTON, G.R., HOFFMAN, E.P., and KUNKEL, L.M. (1989). Conversion of mdx myofibres from dystrophin-negative to positive by injection of normal myoblasts. Nature 337, 176-179.
27. RANDO, T.A., and BLAU, H.M. (1994). Primary mouse myoblast purification, characterization, and transplantation for cell-mediated gene therapy. J. Cell Biol. 125, 1275-1287.
28. RHODES, C.J., and ALARCON, C. (1994). What β-cell defect could lead to hyperproinsulinemia in NIDDM? Diabetes 43, 511-517.
29. ROEBROEK, A.J.M., SHALKEN, J.A., LEUNISSEN, J.A.M., ONNEKINK, C., BLOEMERS, H.P.J., and VAN DE VEN, W.J.M. (1986). Evolutionarily conserved close linkage of the c-fes/fps protooncogene and genetic sequences encoding a receptor-like protein. EMBO J. 5, 2197-2202.
30. SCHAGGER, H., and VON JAGOW, G. (1987). Tricine-sodium dodecyl sulfate-polyacrylamide gel electrophoresis for the separation of proteins in the range from 1 to 100 kDa. Anal. Biochem. 166, 368-379.
31. SCHALKEN, J.A., ROEBROEK, A.J.M., OOMEN, P.P.C.A., WAGENAAR, S.SC., DEDRUYNE, F.M.J., BLOMEMERS, H.P.J., and VAN DE VEN, W.J.M. (1987). fur gene expression as a discriminating marker for small cell and nonsmall cell lung carcinomas. J. Clin. Invest. 80, 1545-1549.
32. SCHWARTZ, G.P., BURKE, G.T., and KATOSOYANNIS, G. (1987). A superactive insulin:[B10-aspartic acid]insulin(human). Proc. Natl. Acad. Sci. USA 84, 6408-6411.
33. SEIDAH, N.G., GASPAR, L., MION, P., MARCINKIEWIEZ, M., MBIKAY, M., and CHRETIEN, M. (1990). cDNA sequence of two distinct pituitary proteins homologous to KEX2 and furin gene products. Tissue-specific mRNAs encoding candidates for pro-hormone processing proteinases. DNA Cell Biol. 9, 415-424.
34. SHOELSON, S.E., LU, Z.-X., PARLAUTAN, L. LYNCH, C.S., and WEISS, M.A. (1992). Mutations at the dimer, hexamer, and receptor-binding surfaces of insulin independently affect insulin-insulin and insulin-receptor interactions. Biochemistry 31, 1757-1767.
35. SMEEKENS, S.P., AVRUCH, A.S., LAMENDOLA, J., CHAN, S.J., and STEINER, D.F. (1991). Identification of a cDNA encoding a second putative prohormone convertase related to PC2 in AtT20 cells and islets of Langerhans. Proc. Natl. Acad. Sci. USA 88, 340-344.
36. SMEEKENS, S.P., MONTAG, A.G., THOMAS, G., ALBIGES-RIZO, C., CARROLL, R., BENIG, M., PHILLIPS, L.A., MARTIN, S., OHAGI, S., GARDNER, P., SWIFT, H.H., and STEINER, D.F. (1992). Proinsulin processing by the subtilisin-related proprotein convertases furin, PC2 and PC3. Proc. Natl. Acad. Sci. USA 89, 8822-8826.
37. SMEEKENS, S.P., and STEINER, D.F. (1990). Identification of a human insulinoma cDNA encoding a novel mammalian protein structurally related to the yeast dibasic processing KEX2. J. Biol. Chem. 265, 2997-3000.
38. STEINER, D.F., SMEEKENS, S.P., OHAGI, S., and CHAN, S.J. (1992). The new enzymology of precursor processing endoproteases. J. Biol. Chem. 267, 23435-23438.
39. WEISS, M.A., HUA, Q.X., LYNCH, C.S., FRANK, B.H., and SHOELSON, S.E. (1991). Heteronuclear 2D NMR studies on an engineered insulin monomer: assignment and characterization of the receptor-binding surface by selective 2H and 13 C labeling with application to protein design. Biochemistry 30, 7373-7389.
40. WOLFF, J.A., LUDTKE, J.J., ACSADI, G., WILLIAMS, P., and JANI, A. (1992). Long-term persistence of plasmid DNA and foreign gene expression in mouse muscle. Hum. Mol. Genet. 1, 363-369.
41. YANAGITA, M., NAKAYAMA, K., and TAKEUCHI, T. (1992). Processing of mutated proinsulin with tetrabasic cleavage sites to bioactive insulin in the non-endocrine cell line, COS-7. FEBS Lett. 311, 55-59.
42. YANAGITA, M., HOSHINO, H., NAKAYAMA, K., and TAKEUCHI, T. (1993). Processing of mutated proinsulin with tetrabasic cleavage sites to mature insulin reflects the expression of furin in nonendocrine cell lines. Endocrinology 133, 639-644.