Salivary glands as a potential gene transfer target for gene therapeutics of some monogenetic endocrine disorders

Journal of Endocrinology

Volumn 185, Issue 3, 2005, Pages 363-372

  Voutetakis, Antonis ^a   Bossis, Ioannis ^a   Kok, Marc R ^a,b   Zhang, Weitian ^a   Wang, Jianghua ^a   Cotrim, Ana P ^a   Zheng, Changyu ^a   Chiorini, John A ^a   Nieman, Lynnette K ^c   Baum, Bruce J ^a  

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

^b ACADEMIC MEDICAL CENTER   (Netherlands)

^c EUNICE KENNEDY SHRIVER NATIONAL INSTITUTE OF CHILD HEALTH AND HUMAN DEVELOPMENT   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; COMPLEMENTARY DNA; DEXAMETHASONE; ERYTHROPOIETIN; GREEN FLUORESCENT PROTEIN; HUMAN GROWTH HORMONE; PARVOVIRUS VECTOR; SECRETORY PROTEIN; VIRUS VECTOR;

ADENOVIRUS 5; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; ENDOCRINE DISEASE; GENE TARGETING; GENE TRANSFER; IMMUNOHISTOCHEMISTRY; MOUSE; MOUSE STRAIN; NONHUMAN; PRIORITY JOURNAL; PROTEIN LOCALIZATION; SALIVARY GLAND; TISSUE SECTION; TRANSGENE; VIRAL GENE THERAPY; VIRUS RECOMBINANT;

ADENOVIRIDAE; ANIMALS; DEPENDOVIRUS; ERYTHROPOIETIN; GENE EXPRESSION; GENE THERAPY; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; HORMONE REPLACEMENT THERAPY; HUMAN GROWTH HORMONE; HUMANS; IMMUNOHISTOCHEMISTRY; MICE; MICE, INBRED BALB C; MICROSCOPY, FLUORESCENCE; SALIVA; SUBMANDIBULAR GLAND; TRANSDUCTION, GENETIC; TRANSGENES;

EID: 21244443038     PISSN: 00220795     EISSN: None     Source Type: Journal    
DOI: 10.1677/joe.1.06171     Document Type: Article

References (45)

1. Adesanya MR, Redman RS, Baum BJ & O'Connell BC 1996 Immediate inflammatory responses to adenovirus-mediated gene transfer in rat salivary glands. Human Gene Therapy 7 1085-1093.
2. Almazan F, Gonzalez JM, Penzes Z, Izeta A, Calvo E, Plana-Duran J & Enjuanes L 2000 Engineering the largest RNA virus genome as an infectious bacterial artificial chromosome. PNAS 97 5516-5521.
3. Amerongen AV & Veerman EC 2002 Saliva - the defender of the oral cavity. Oral Diseases 8 12-22.
4. Auricchio A, O'Connor E, Weiner D, Gao GP, Hildinger M, Wang L, Calcedo R & Wilson JM 2002 Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins. Journal of Clinical Investigation 110 499-504.
5. Barzon L, Bonaguro R, Palu G & Boscaro M 2000 New perspectives for gene therapy in endocrinology. European Journal of Endocrinology 143 447-466.
6. Baum BJ, Berkman ME, Marmary Y, Goldsmith CM, Baccaglini L, Wang S, Wellner RB, Hoque AT, Atkinson JC, Yamagishi H, Kagami H, Parlow AF & Chao J 1999 Polarized secretion of transgene products from salivary glands in vivo. Human Gene Therapy 10 2789-2797.
7. Baum BJ, Wellner RB & Zheng C 2002 Gene transfer to salivary glands. International Review of Cytology 213 93-146.
8. Bazan JF 1989 A novel family of growth factor receptors: a common binding domain in the growth hormone, prolactin, erythropoietin and IL-6 receptors, and the p75 IL-2 receptor beta-chain. Biochemical and Biophysical Research Communications 164 788-795.
9. Bohl D, Bosch A, Cardona A, Salvetti A & Heard JM 2000 Improvement of erythropoiesis in beta-thalassemic mice by continuous erythropoietin delivery from muscle. Blood 95 2793-2798.
10. Carroll RJ, Hammer RE, Chan SJ, Swift HH, Rubenstein AH & Steiner DF 1988 A mutant human proinsulin is secreted from islets of Langerhans in increased amounts via an unregulated pathway. PNAS 85 8943-8947.
11. Castle D & Castle A 1998 Intracellular transport and secretion of salivary proteins. Critical Reviews in Oral Biology and Medicine 9 4-22.
12. Chan SJ, Seino S, Gruppuso PA, Schwartz R & Steiner DF 1987 A mutation in the B chain coding region is associated with impaired proinsulin conversion in a family with hyperproinsulinemia. PNAS 84 2194-2197.
13. Chao H, Monahan PE, Liu Y, Samulski RJ & Walsh CE 2001 Sustained and complete phenotype correction of hemophilia B mice following intramuscular injection of AAV1 serotype vectors. Molecular Therapy 4 217-222.
14. Chiorini JA, Wendtner CM., Urcelay E, Safer B, Hallek M & Kotin RM 1995 High-efficiency transfer of the T cell co-stimulatory molecule B7-2 to lymphoid cells using high-titer recombinant adeno-associated virus vectors. Human Gene Therapy 6 1531-1541.
15. Cook DI, Van Lennep EW, Roberts ML & Young JA 1994 Secretion by the major salivary glands. In Physiology of the gastrointestinal tract. 3rd edition, pp 1016-1117. Ed LR Johnson. New York: Raven Press.
16. Crystal RG 1995 The gene as a drug. Nature Medicine 1 15-17.
17. Dannies P 2002 Mechanisms for storage of prolactin and growth hormone in secretory granules. Molecular Genetics and Metabolism 76 6-13.
18. Dube S, Fisher JW & Powell JS 1988 Glycosylation at specific sites of erythropoietin is essential for biosynthesis, secretion, and biological function. Journal of Biological Chemistry 263 17516-17521.
19. Gerdes HH & Glombik MM 1999 Signal-mediated sorting to the regulated pathway of protein secretion. Anatomischer Anzeiger 181 447-453.
20. Goldspink G 2003 Skeletal muscle as an artificial endocrine tissue. Best Practice and Research Clinical Endocrinology and Metabolism 17 211-222.
21. Harding TC, Koprivnikar KE, Tu GH, Zayek N, Lew S, Subramanian A, Sivakumaran A, Frey D, Ho K, VanRoey MJ, Nichols TC, Bellinger DA, Yendluri S, Waugh J, McArthur J, Veres G & Donahue BA 2004 Intravenous administration of an AAV-2 vector for the expression of factor IX in mice and a dog model of hemophilia B. Gene Therapy 2004 204-213.
22. He X, Goldsmith CM, Marmary Y, Wellner RB, Parlow AF, Nieman LK & Baum BJ 1998 Systemic action of human growth hormone following adenovirus-mediated gene transfer to rat submandibular glands. Gene Therapy 5 537-541.
23. Hoque AT, Baccaglini L & Baum BJ 2001 Hydroxychloroquine enhances the endocrine secretion of adenovirus-directed growth hormone from rat submandibular gland in vivo. Human Gene Therapy 12 1333-1341.
24. Isenman L, Liebow C & Rothman S 1999 The endocrine secretion of mammalian digestive enzymes by exocrine glands. American Journal of Physiology 276 E223-E232.
25. Johnston J, Tazelaar J, Rivera VM, Clackson T, Gao GP & Wilson JM 2003 Regulated expression of erythropoietin from an AAV vector safely improves the anemia of beta-thalassemia in a mouse model. Molecular Therapy 7 493-497.
26. Kagami H, Atkinson JC, Michalek SM, Handelman B, Yu S, Baum BJ & O'Connell BC 1998 Repetitive adenovirus administration to the parotid gland: role of immunological barriers and induction of oral tolerance. Human Gene Therapy 9 305-313.
27. Kaludov N, Handelman B & Chiorini JA 2002 Scalable purification of adeno-associated virus type 2, 4, or 5 using ion-exchange chromatography. Human Gene Therapy 13 1235-1243.
28. Kay MA, Glorioso JC & Naldini L 2001 Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics. Nature Medicine 7 33-40.
29. Kelly RB 1985 Pathways of protein secretion in eukaryotes. Science 230 25-32.
30. Kendall RG 2001 Erythropoietin. Clinical and Laboratory Haematology 23 71-80.
31. Kok MR, Yamano S, Lodde BM, Wang J, Couwenhoven RI, Yakar S, Voutetakis A, Leroith D, Schmidt M, Afione S, Pillemer SR, Tsutsui MT, Tak PP, Chiorini JA & Baum BJ 2003 Local adeno-associated virus-mediated interleukin 10 gene transfer has disease-modifying effects in a murine model of Sjogren's syndrome. Human Gene Therapy 14 1605-1618.
32. Lai CM, Lai YK & Rakoczy PE 2002 Adenovirus and adeno-associated virus vectors. DNA and Cell Biology 21 895-913.
33. Loh YP, Maldonado A, Zhang C, Tam WH & Cawley N 2002 Mechanisms of sorting pro-opiomelanocortin and pro-enkephalin to the regulated secretory pathway of neuroendocrine cells. Annals of the New York Academy of Sciences 971 416-425.
34. Marmorstein AD, Csaky KG, Baffi J, Lam L, Rahaal F & Rodriguez-Boulan 2000 Saturation of, and competition for entry into, the apical secretory pathway. PNAS 97 3248-3253.
35. Mittereder N, March KL & Trapnell BC 1996 Evaluation of the concentration and bioactivity of adenovirus vectors for gene therapy. Journal of Virology 70 7498-7509.
36. Mujais SK, Beru N, Pullman TN & Goldwasser E 1999 Erythropoietin is produced by tubular cells of the rat kidney. Cell Biochemistry and Biophysics 30 153-166.
37. Samakoglu S, Bohl D & Heard JM 2002 Mechanisms leading to sustained reversion of beta-thalassemia in mice by doxycycline-controlled Epo delivery from muscles. Molecular Therapy 6 793-803.
38. Snyder RO, Miao CH, Patijn GA, Spratt SK, Danos O, Nagy D, Gown AM, Winther B, Meuse L, Cohen LK, Thompson AR & Kay MA 1997 Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nature Genetics 16 270-276.
39. Voutetakis A, Wang J & Baum BJ 2004a Utilizing endocrine secretory pathways in salivary glands for systemic gene therapeutics. Journal of Cellular Physiology 199 1-7.
40. Voutetakis A, Kok MR, Zheng C, Bossis I, Wang J, Cotrim AP, Marracino N, Goldsmith CM, Chiorini JA, Loh YP, Nieman LK & Baum BJ 2004b Re-engineered salivary glands are stable endogenous bioreactors for systemic gene therapeutics. PNAS 101 3053-3058.
41. Wang J, Voutetakis A, Zheng C & Baum BJ 2004 Rapamycin control of exocrine protein levels in saliva after adenoviral vector-mediated gene transfer. Gene Therapy 11 729-733.
42. Wang J, Cawley NX, Voutetakis A, Rodriguez YM, Goldsmith CM, Nieman LK, Hoque S, Frank SJ, Snell CR, Loh YP & Baum BJ 2005 Partial re-direction of transgenic human growth hormone secretion from rat salivary glands. Human Gene Therapy (in press).
43. Yamano S, Huang LY, Ding C, Chiorini JA, Goldsmith CM, Wellner RB, Golding B, Kotin RM, Scott DE & Baum BJ 2002 Recombinant adeno-associated virus serotype 2 vectors mediate stable interleukin 10 secretion from salivary glands into the bloodstream. Human Gene Therapy 13 287-298.
44. Zheng C, Hoque AT, Braddon VR, Baum BJ & O'Connell BC 2001 Evaluation of salivary gland acinar and ductal cell-specific promoters in vivo with recombinant adenoviral vectors. Human Gene Therapy 12 2215-2223.
45. Zufferey R & Aebischer P 2004 Salivary glands and gene therapy: the mouth waters. Gene Therapy 11 1425-1426.