Long-term human growth hormone expression and partial phenotypic correction by plasmid-based gene therapy in an animal model of isolated growth hormone deficiency

Journal of Gene Medicine

Volumn 12, Issue 7, 2010, Pages 580-585

  Oliveira, Nélio A J ^a   Cecchi, Cláudia R ^a   Higuti, Eliza ^a   Oliveira, João E ^a   Jensen, Thomas G ^b   Bartolini, Paolo ^a   Peroni, Cibele N ^a  

^a INSTITUTO DE PESQUISAS ENERGÉTICAS E NUCLEARES   (Brazil)

^b AARHUS UNIVERSITY   (Denmark)

Author keywords

Electroporation; Gene therapy; Human growth hormone; Little mice; Naked DNA; Nonviral vector

Indexed keywords

DNA; PLASMID DNA; RECOMBINANT GROWTH HORMONE; UBIQUITIN C; HUMAN GROWTH HORMONE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; DOSE RESPONSE; ELECTROPORATION; GENE SEQUENCE; GENE THERAPY; GROWTH HORMONE BLOOD LEVEL; GROWTH HORMONE DEFICIENCY; IMMUNOCOMPETENCE; MOUSE; NONHUMAN; PHENOTYPE; PLASMID; PRIORITY JOURNAL; QUADRICEPS FEMORIS MUSCLE; SCID MOUSE; TREATMENT DURATION; WEIGHT GAIN; ANIMAL; BODY WEIGHT; C57BL MOUSE; DEFICIENCY; DISEASE MODEL; DWARFISM, PITUITARY; GENETICS; HUMAN; METABOLISM; MUSCLE; ORGAN SIZE; PATHOLOGY; TIME;

ANIMALS; BODY WEIGHT; DISEASE MODELS, ANIMAL; DWARFISM, PITUITARY; GENETIC THERAPY; HUMAN GROWTH HORMONE; HUMANS; MICE; MICE, INBRED C57BL; MUSCLES; ORGAN SIZE; PHENOTYPE; PLASMIDS; TIME FACTORS;

EID: 77955600899     PISSN: 1099498X     EISSN: 15212254     Source Type: Journal    
DOI: 10.1002/jgm.1470     Document Type: Article

References (49)

1. Ribela MTCP, Gout PW, Bartolini P. Synthesis and chromatographic purification of recombinant human pituitary hormones. J Chromatogr B 2003; 790: 285-316. (Pubitemid 36588789)
2. Butler M. Animal cell cultures: recent achievements and perspectives in the production of biopharmaceuticals. Appl Microbiol Biotechnol 2005; 68: 283-291. (Pubitemid 41159998)
3. Walsh G. Biopharmaceutical benchmarks. Nature Biotechnol 2006; 24: 769-776.
4. Leader B, Baca QJ, Golan DE. Protein therapeutics: a summary and pharmacological classification. Nat Rev Drug Discov 2008; 7: 21-39.
5. Taichman LB. Systemic replacement therapy from genetically modified epidermal keratinocytes. Proc Assoc Am Physicians 1999; 111: 206-210.
6. Peroni CN, Cecchi CR, Rosauro CW, et al. Secretion of mouse growth hormone by transduced primary human keratinocytes: prospects for an animal model of cutaneous gene therapy. J Gene Med 2008; 10: 734-743.
7. Ehrhardt A, Haase R, Schepers A, et al. Episomal vectors for gene therapy. Curr Gene Ther 2008; 8: 147-161.
8. Teumer J, Lindahl A, Green H. Human growth hormone in the blood of athymic mice grafted with cultures of hormone-secreting human keratinocytes. FASEB J 1990; 4: 3245-3250. (Pubitemid 120012571)
9. Jensen UB, Jensen TG, Jensen PKA, et al. Gene transfer into cultured human epidermis and its transplantation onto immunodeficient mice: an experimental model for somatic gene therapy. J Invest Dermatol 1994; 103: 391-394.
10. Peroni CN, Cecchi CR, Damiani R, et al. High-level secretion of growth hormone by retrovirally transduced primary human keratinocytes: prospects for an animal model of cutaneous gene therapy. Mol Biotechnol 2006; 34: 239-245. (Pubitemid 46030397)
11. Peroni CN, Gout PW, Bartolini P. Animal models for growth hormone gene therapy. Curr Gene Ther 2005; 5: 493-509.
12. White SJ, Page SM, Margaritis P, et al. Long-term expression of human clotting factor IX from retrovirally transduced primary human keratinocytes in vivo. Hum Gene Ther 1998; 9: 1187-1195.
13. Sondergaard M, Dagnaes-Hansen F, Flyvbjerg A, et al. Normalization of growth in hypophysectomized mice using hydrodynamic transfer of the human growth hormone gene. Am J Physiol Endocrinol Metab 2003; 285: E427-E432.
14. Eicher EM, Beamer WG. Inherited ateolitic dwarfism in mice. Characteristics of the mutation, little, on chromosome 6. J Hered 1976; 67: 87-91.
15. Godfrey P, Rahal JO, Beamer WG, Copeland NG, Jenkins NA, Mayo KE. GHRH receptor of little mice contains a missense mutation in the extracellular domain that disrupts receptor function. Nat Genet 1993; 4: 227-232. (Pubitemid 23205169)
16. Lin SC, Lin CR, Gukovsky I, Lusis AJ, Sawchenko PE, Rosenfeld, MG. Molecular basis of the little mouse phenotype and implications for cell type-specific growth. Nature 1993; 364: 208-213.
17. Jansson JO, Downs TR, Beamer WG, et al. Receptor-associated resistance to growth hormone-releasing factor in dwarf 'little' mice. Science 1986; 232: 511-512.
18. Schuler W, Bosma MJ. Nature of the scid defect: a defective VDJ recombinase system. Curr Top Microbiol Immunol 1989; 152: 55-62.
19. Yang X, Beamer WG, Huynh H, Pollak M. Reduced growth of human breast cancer xenografts in hosts homozygous for the lit mutation. Cancer Res 1996; 56: 1509-1511.
20. Tripathy SK, Svensson EC, Black HB, et al. Long-term expression of erythropoietin in the systemic circulation of mice after intramuscular injection of a plasmid DNA vector. Proc Natl Acad Sci USA 1996; 93: 10876-10880.
21. MacColl GS, Novo FJ,Marshall NJ, et al.Optimization of growth hormone production by muscle cells using plasmid DNA. J Endocrinol 2000; 165: 329-336.
22. Fattori E, Cappelletti M, Zampaglione I, et al. Gene electrotransfer of an improved erythropoietin plasmid in mice and non-human primates. J Gene Med 2005; 7: 228-236. (Pubitemid 40348027)
23. Ratanamart J, Shaw JA. Plasmid-mediated muscle-targeted gene therapy for circulating therapeutic protein replacement: a tale of the tortoise and the hare? Curr Gene Ther 2006; 6: 93-110.
24. Prud'homme GJ, Draglia-Akli R, Wang Q. Plasmid-based gene therapy of diabetes mellitus. Gene Ther 2007; 14: 553-564.
25. Deitel K, Dantzer D, Ferguson P, et al. Reduced growth of human sarcoma xenografts in hosts homozygous for the lit mutation. J Surg Oncol 2002; 81: 75-79.
26. Dagnaes-Hansen F, Holst HU, Sondergaard M, et al. Physiological effects of human growth hormone produced after hydrodynamic gene transfer of a plasmid vector containing the human ubiquitin promotor. J Mol Med 2002; 80: 665-670.
27. Bellini MH, Peroni CN, Bartolini P. Increases in weight of growth hormone-deficient and immunodeficient (lit/scid) dwarf mice after grafting of hGH-secreting, primary human keratinocytes. FASEB J 2003; 17: 2322-2324.
28. Bellini MH, Bartolini P. In vivo bioassay for the potency determination of human growth hormone in dwarf 'little' mice. Endocrinology 1993; 132: 2051-2055.
29. Bellini MH, Mathor MB, De Luca M, et al. Ultrasensitive in vivo bioassay detects bioactive human growth hormone in transduced primary human keratinocytes. J Endocrinol Invest 1998; 21: 1-6.
30. Golzio M, Rols MP, Teissié J. In vitro and in vivo electric field-mediated permeabilization, gene transfer, and expression. Methods 2004; 33: 126-135. (Pubitemid 38578416)
31. Hammer RE, Palmiter, RD, Brinster RL. Partial correction of murine hereditary growth disorder by germ-line incorporation of a new gene. Nature 1984; 311: 65-67.
32. Hahn TM, Copeland KC, Woo SL. Phenotypic correction of dwarfism by constitutive expression of growth hormone. Endocrinology 1996; 137: 4988-4993.
33. Anwer K, Shi M, French MF, et al. Systemic effect of human growth hormone after intramuscular injection of a single dose of a muscle-specific gene medicine. Hum Gene Ther 1998; 9: 659-670.
34. Draghia-Akli R, Li XG, Schwartz, RJ. Enhanced growth by ectopic expression of growth hormone releasing hormone using an injectable myogenic vector. Nature Biotechnol 1997; 15: 1285-1289.
35. Draghia-Akli R, Fiorotto ML, Hill LA, et al. Myogenic expression of an injectable protease-resistant growth hormone-releasing hormone augments long-term growth in pigs. Nature Biotechnol 1999; 17: 1179-1183.
36. Draghia-Akli R, Hahn KA, King GK, et al. Effects of plasmid-mediated growth hormone-releasing hormone in severely debilitated dogs with cancer. Mol Ther 2002; 6: 830-836. (Pubitemid 36054316)
37. Draghia-Akli R, Malone PB, Hill LA, et al. Enhanced animal growth via ligand-regulated GHRH myogenic-injectable vectors. FASEB J 2002; 16: 426-428.
38. Draghia-Akli R, Pope MA, Brown PA, et al. Plasmid-based expression technology using growth hormone releasing hormone: a novel method for physiologically stimulating long-term growth hormone secretion. Comb Chem High Throughput Screen 2006; 9: 181-185.
39. Brown PA, Bodles-Brakhop AM, Pope MA, et al. Gene therapy by electroporation for the treatment of chronic renal failure in companion animals. BMC Biotechnol 2009; 9: 4.
40. Wilson DB, Wyatt DP, Gadler RM, et al. Quantitative aspects of growth hormone cell maturation in the normal and little mutant mouse. Acta Anat 1988; 131: 150-155.
41. Juul A, Hjortskov N, Jepsen LT, et al. Growth-hormone deficiency and hyperthermia during exercise - a controlled-study of 16 GH-deficient patients. J Clin Endocrinol Metab 1995; 80: 3335-3340.
42. Becker BA, Klir JJ, Matteri RL, et al. Endocrine and thermoregulatory responses to acute thermal exposures in 6-month-old pigs reared in different neonatal environments. J Therm Biol 1997; 22: 87-93.
43. Hana V, Prasny M, Marek J, et al. Reduced microvascular perfusion and reactivity in adult GH deficient patients is restored by GH replacement. Eur J Endocrinol 2002; 147: 333-337.
44. Gibney J, Healy Ml, Sonksen PH. The growth hormone/insulin-like growth factor-I axis in exercise and sport. Endocr Rev 2007; 28: 603-624.
45. Khan AS, Draghia-Akli R, Shypailo RJ, Ellis K, Mersman H, Fiorotto ML. A comparison of the growth responses following intramuscular GHRH plasmid administration vs. daily growth hormone injections in young pigs. Mol Ther 2010; 18: 327-333.
46. Melman A, Bar-Chama N, McCullough A, et al. Plasmid-based gene transfer for treatment of erectile dysfunction and overactive bladder: results of a phase I trial. Isr Med Assoc J 2007; 9: 143-146. (Pubitemid 46470391)
47. Baumgartner I, Chronos N, Comerota A, et al. Local gene transfer and expression following intramuscular administration of FGF-1 plasmid DNA in patients with critical limb ischemia. Mol Ther 2009; 17: 914-921.
48. Christ GJ, Andersson K, Williams K, et al. Smooth-muscle-specific gene transfer with the human Maxi-K channel improves erectile function and enhances sexual behavior in atherosclerotic cynomolgus monkeys. Eur Urol 2009; 56: 1055-1066.
49. Wallace M, Evans B, Woods S, et al. Tolerability of two sequential electroporation treatments using MedPulser DNA delivery system (DDS) in healthy adults. Mol Ther 2009; 17: 922-928.