Transgenic α-1-antitrypsin secreted into the bloodstream from salivary glands is biologically active

Oral Diseases

Volumn 17, Issue 5, 2011, Pages 476-483

  Perez, P ^a   Adriaansen, J ^a   Goldsmith, C M ^a   Zheng, C ^a   Baum, B J ^a  

^a NATIONAL INSTITUTE OF DENTAL AND CRANIOFACIAL RESEARCH   (United States)

Author keywords

Bioactivity; Gene therapy; Human 1 antitrypsin; Salivary glands; Secretion

Indexed keywords

ADENOVIRUS VECTOR; ALPHA 1 ANTITRYPSIN; ENDOGLYCOSIDASE H; GLYCOSIDASE; LEUKOCYTE ELASTASE; N ACETYL BETA GLUCOSAMINIDASE; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; BLOOD FLOW; CELLULAR DISTRIBUTION; CONTROLLED STUDY; ENZYME INHIBITION; ENZYME LINKED IMMUNOSORBENT ASSAY; GENE TRANSFER; GLYCOSYLATION; IMMUNOHISTOCHEMISTRY; MALE; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROTEIN EXPRESSION; PROTEIN SECRETION; PROTEIN SYNTHESIS; RAT; SALIVARY GLAND; SEROTYPE; SUBMANDIBULAR GLAND;

ADENOVIRIDAE; ALPHA 1-ANTITRYPSIN; ANIMALS; CELL LINE; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; GLYCOSIDE HYDROLASES; GLYCOSYLATION; HUMANS; IMMUNOHISTOCHEMISTRY; LEUKOCYTE ELASTASE; MALE; MICE; MICE, INBRED BALB C; PEPTIDE-N4-(N-ACETYL-BETA-GLUCOSAMINYL) ASPARAGINE AMIDASE; PLASMIDS; RATS; RATS, WISTAR; SALIVA; SERINE PROTEINASE INHIBITORS; SUBMANDIBULAR GLAND; TISSUE EXTRACTS;

EID: 79957950798     PISSN: 1354523X     EISSN: 16010825     Source Type: Journal    
DOI: 10.1111/j.1601-0825.2010.01775.x     Document Type: Article

References (32)

1. Adriaansen J, Perez P, Goldsmith CM, Zheng C, Baum BJ (2008). Differential sorting of human parathyroid hormone after transduction of mouse and rat salivary glands. Hum Gene Ther 19: 1021-1028.
2. Amalfitano A, Chamberlain JS (1997). Isolation and characterization of packaging cell lines that coexpress the adenovirus E1, DNA polymerase, and preterminal proteins: implications for gene therapy. Gene Ther 4: 258-263.
3. Balfour-Lynn IM (1999). The protease-antiprotease battle in the cystic fibrotic lung. JR Soc Med 92(Suppl. 37): 23-30.
4. Baum BJ, Berkman ME, Marmary Y et al (1999). Polarized secretion of transgene products from salivary glands in vivo. Hum Gene Ther 10: 2789-2797.
5. Baum BJ, Wellner RB, Zheng C (2002). Gene transfer to salivary glands. Int Rev Cytol 213: 93-146.
6. Baum BJ, Voutetakis A, Wang J (2004). Salivary glands: novel target sites for gene therapeutics. Trends Mol Med 10: 585-590.
7. Becker TC, Noel RJ, Coats WS et al (1994). Use of recombinant adenovirus for metabolic engineering of mammalian cells. Methods Cell Biol 43(Pt A): 161-189.
8. Cotrim AP, Sowers A, Mitchell JB, Baum BJ (2007). Prevention of irradiation-induced salivary hypofunction by microvessel protection in mouse salivary glands. Mol Ther 15: 2101-2106.
9. Egelund R, Rodenburg KW, Andreasen PA, Rasmussen MS, Guldberg RE, Petersen TE (1998). An ester bond linking a fragment of a serine protease to its serpin inhibitor. Biochemistry 37: 6375-6379.
10. Fregonese L, Stolk J (2008). Hereditary alpha-1-antitrypsin deficiency and its clinical consequences. Orphanet J Rare Dis 3: 16.
11. Gooptu B, Lomas DA (2009). Conformational pathology of the serpins: themes, variations, and therapeutic strategies. Annu Rev Biochem 78: 147-176.
12. He X, Goldsmith CM, Marmary Y et al (1998a). Systemic action of human growth hormone following adenovirus-mediated gene transfer to rat submandibular glands. Gene Ther 5: 537-541.
13. He X, Kuijpers GA, Goping G et al (1998b). A polarized salivary cell monolayer useful for studying transepithelial fluid movement in vitro. Pflugers Arch 435: 375-381.
14. Kagami H, O'Connell BC, Baum BJ (1996). Evidence for the systemic delivery of a transgene product from salivary glands. Hum Gene Ther 7: 2177-2184.
15. Kohnlein T, Welte T (2008). Alpha-1 antitrypsin deficiency: pathogenesis, clinical presentation, diagnosis, and treatment. Am J Med 121: 3-9.
16. Kok MR, Yamano S, Lodde BM et al (2003). Local adeno-associated virus-mediated interleukin 10 gene transfer has disease-modifying effects in a murine model of Sjögren's syndrome. Hum Gene Ther 14: 1605-1618.
17. Kwon KS, Yu MH (1997). The effect of glycosylation on the stability of alpha1-antitrypsin toward urea denaturation and thermal deactivation. Biochim Biophys Acta 1335: 265-272.
18. Lodde BM, Mineshiba F, Wang J et al (2006). Effect of vasoactive intestinal peptide gene transfer in a murine model of Sjögren's syndrome. Ann Rheum Dis 65: 195-200.
19. Lomas DA, Carrell RW (1993). A protein structural approach to the solution of biological problems: α1-antitrypsin as a recent example. Am J Physiol 265: L211-L219.
20. Maley F, Trimble RB, Tarentino AL, Plummer TH Jr (1989). Characterization of glycoproteins and their associated oligosaccharides through the use of endoglycosidases. Anal Biochem 180: 195-204.
21. Mills K, Mills PB, Clayton PT, Mian N, Johnson AW, Winchester BG (2003). The underglycosylation of plasma alpha 1-antitrypsin in congenital disorders of glycosylation type I is not random. Glycobiology 13: 73-85.
22. Morgan K, Kalsheker NA (1997). Regulation of the serine protease inhibitor (SERPIN) gene alpha 1-antitrypsin: a paradigm for other SERPINs. Int J Biochem Cell Biol 29: 1501-1511.
23. Perez P, Rowzee AM, Zheng C, Adriaansen J, Baum BJ (2010). Salivary epithelial cells: an unassuming target site for gene therapeutics. Int J Biochem Cell Biol 42: 773-777.
24. Rothman JE, Fine RE (1980). Coated vesicles transport newly synthesized membrane glycoproteins from endoplasmic reticulum to plasma membrane in two successive stages. Proc Natl Acad Sci USA 77: 780-784.
25. Sidhar SK, Lomas DA, Carrell RW, Foreman RC (1995). Mutations which impede loop/sheet polymerization enhance the secretion of human alpha 1-antitrypsin deficiency variants. J Biol Chem 270: 8393-8396.
26. Sugito T, Mineshiba F, Zheng C, Cotrim AP, Goldsmith CG, Baum BJ (2009). Transient TWEAK overexpression leads to a general salivary epithelial proliferation. Oral Dis 15: 76-81.
27. Sun Z, Yang P (2004). Role of imbalance between neutrophil elastase and α1-antitrypsin in cancer development and progression. Lancet Oncol 5: 182-190.
28. Voutetakis A, Kok MR, Zheng C et al (2004). Reengineered salivary glands are stable endogenous bioreactors for systemic gene therapeutics. Proc Natl Acad Sci USA 101: 3053-3058.
29. Voutetakis A, Zheng C, Metzger M et al (2008). Sorting of transgenic secretory proteins in rhesus macaque parotid glands after adenovirus-mediated gene transfer. Hum Gene Ther 19: 1401-1405.
30. Voutetakis A, Cotrim AP, Rowzee A et al (2010). Systemic delivery of bioactive glucagon-like peptide 1 following adenoviral mediated gene transfer in murine salivary gland. Endocrinology 151: 4566-4572.
31. Zheng C, Baum BJ, Iadarola MJ, O'Connell BC (2000). Genomic integration and gene expression by a modified adenoviral vector. Nat Biotechnol 18: 176-180.
32. Zheng C, Cotrim AP, Sunshine AN et al (2009). Prevention of radiation-induced oral mucositis after adenoviral vector-mediated transfer of the keratinocyte growth factor cDNA to mouse submandibular glands. Clin Cancer Res 15: 4641-4648.