Clinically applicable procedure for gene delivery to fetal gut by ultrasound-guided gastric injection: Toward prenatal prevention of early-onset intestinal diseases

Human Gene Therapy

Volumn 17, Issue 7, 2006, Pages 767-779

  David, A L ^a   Peebles, D M ^a   Gregory, L ^b   Waddington, S N ^b   Themis, M ^b   Weisz, B ^a   Ruthe, A ^a   Lawrence, L ^b   Cook, T ^b   Rodeck, C H ^a   Coutelle, C ^b  

^a ROYAL FREE AND UNIVERSITY COLLEGE MEDICAL SCHOOL   (United Kingdom)

^b IMPERIAL COLLEGE SCHOOL OF MEDICINE   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

2 DIETHYLAMINOETHANOL; 5 BROMO 4 CHLORO 3 INDOLYL BETA DEXTRO GALACTOPYRANOSIDE; ADENOVIRUS VECTOR; BETA GALACTOSIDASE; DECANOATE SODIUM; DEXTRAN; FATTY ACID; FLUOROCARBON; GALACTOSE; PERFLUOROOCTYL BROMIDE; PYRANOSIDE; TRANSMEMBRANE CONDUCTANCE REGULATOR; UNCLASSIFIED DRUG;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; COMPLEX FORMATION; CONGENITAL DISORDER; CONTROLLED STUDY; CYSTIC FIBROSIS; ECHOGRAPHY; ENTEROPATHY; ENZYME LINKED IMMUNOSORBENT ASSAY; FEMALE; FETAL THERAPY; FETUS; GENE TARGETING; GENE TRANSFER; GENETIC TRANSDUCTION; GESTATIONAL AGE; HUMAN; HUMAN CELL; IMMUNOHISTOCHEMISTRY; INJECTION; INTESTINE EPITHELIUM; INTESTINE VILLUS; MECONIUM ILEUS; NONHUMAN; PROTEIN EXPRESSION; REPORTER GENE; SHEEP; STOMACH EPITHELIUM; TIGHT JUNCTION; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY;

ADENOVIRIDAE; ANIMALS; BETA-GALACTOSIDASE; CYSTIC FIBROSIS; FEMALE; FETOSCOPY; FETUS; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENES, REPORTER; GENETIC VECTORS; INTESTINAL DISEASES; INTESTINES; SHEEP; STOMACH; TISSUE DISTRIBUTION;

ADENOVIRIDAE; OVIS ARIES;

EID: 33746218856     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2006.17.767     Document Type: Article

References (48)

1. ARCASOY, S.M., LATOCHE, J.D., GONDOR, M., PITT, B.R., and PILEWSKI, J.M. (1997). Polycations increase the efficiency of adenovirus-mediated gene transfer to epithelial and endothelial cells in vitro. Gene Ther. 4, 32-38.
2. ARENAS, R.B., FICHERA, A., MOK, P., BLANCO, M.C., and MICHELASSI, F. (1996). Introduction of human adenomatous polyposis coli gene into Min mice via cationic liposomes. Surgery 120, 712-717.
3. ARSENAULT, P., and MENARD, D. (1987). Cell proliferation in developing human jejunum. Biol. Neonate 51, 297-304.
4. ARSENAULT, P., and MENARD, D. (1989). Cell proliferation during morphogenesis of the human colon. Biol. Neonate 55, 137-142.
5. BARBERA, A., JONES, D.W., ZERBE, G.O., HOBBINS, J.C., BATTAGLIA, F.C., and MESCHIA, G. (1995). Ultrasonographic assessment of fetal growth: Comparison between human and ovine fetus. Am. J. Obstet. Gynecol. 173, 1765-1769.
6. BOUÉ, A., MULLER, F., NEZELOF, C., OURY, J.F., DUCHATEL, F., DUMEZ, Y., AUBRY, M.C., and BOUE, J. (1986). Prenatal diagnosis in 200 pregnancies with a 1-in-4 risk of cystic fibrosis. Hum. Genet. 74, 288-297.
7. BOYLE, M.P., ENKE, R.A., ADAMS, R.J., GUGGINO, W.B., and ZEITLIN, P.L. (2001). In utero AAV-mediated gene transfer to rabbit pulmonary epithelium. Mol. Ther. 4, 115-121.
8. CHARTIER, C., DEGRYSE, E., GANTZER, M., DIETERLE, A., PAVIRANI, A., and MEHTALI, M. (1996). Efficient generation of recombinant adenovirus vectors by homologous recombination in Escherichia coli. J. Virol. 70, 4805-4810.
9. CHENG, D.Y., KOLLS, J.K., LEI, D., and NOEL, R.A. (1997). In vivo and in vitro gene transfer and expression in rat intestinal epithelial cells by E1-deleted adenoviral vector. Hum. Gene Ther. 8, 755-764.
10. CHENG, H., and LEBLOND, C.P. (1974). Origin, differentiation and renewal of the four main epithelial cell types in the mouse small intestine. I-III. Am. J. Anat. 141, 461-520.
11. COLONY, P.C., and NEUTRA, M.R. (1985). Macromolecular transport in the fetal rat intestine. Gastroenterology 89, 294-306.
12. DAVID, A.L., COOK, T., WADDINGTON, S., PEEBLES, D., NIVSARKAR, M., KNAPTON, H., MIAH, M., DAHSE, T., NOAKES, D., SCHNEIDER, H., RODECK, C., COUTELLE, C., and THEMIS, M. (2003). Ultrasound guided percutaneous delivery of adenoviral vectors encoding the β-galactosidase and human factor IX genes to early gestation fetal sheep in utero. Hum. Gene Ther. 14, 353-364.
13. DOUAR, A.-M., ADEBAKIN, S., THEMIS, M., PAVIRANI, A., COOK, T., and COUTELLE, C. (1997). Foetal gene delivery in mice by intra-amniotic administration of retroviral producer cells and adenovirus. Gene Ther. 4, 883-890.
14. DRAY, X., BIENVENU, T., DESMAZES-DUFEU, N., DUSSER, D., MARTEAU, P., and HUBERT, D. (2004). Distal intestinal obstruction syndrome in adults with cystic fibrosis. Clin. Gastroenterol. Hepatol. 2, 498-503.
15. FOREMAN, P.K., WAINWRIGHT, M.J., ALICKE, B., KOVESDI, I., WICKHAM, T.J., SMITH, J.G., MEIER-DAVIS, S., FIX, J.A., DADDONA, P., GARDNER, P., and HUANG, M.T. (1998). Adenovirus-mediated transduction of intestinal cells in vivo. Hum. Gene Ther. 9, 1313-1321.
16. GARRETT, D.J., LARSON, J.E., DUNN, D., MARRERO, L., and COHEN, J.C. (2003). In utero recombinant adeno-associated virus gene transfer in mice, rats, and primates. BMC Biotechnol. 3, 16.
17. GOULET, O., and RUEMMELE, F. (2006). Causes and management of intestinal failure in children. Gastroenterology 130(2 Suppl. 1), S16-S28.
18. GRAHAM, F.L., and VAN DER EB, A.J. (1973). A new technique for the assay of infectivity of human adenovirus 5 DNA. Virology 52, 456-457.
19. HAMILTON, T.E. (1997). Adenoviral-mediated gene transfer to murine small intestine is more efficient in neonates than adults. J. Pediatr. Surg. 32, 373-377.
20. HILL, R.R., and DE BRUYN, G. (1991). Changes in lectin-binding patterns during late fetal development of the rat colon. Acta Anat. 141, 245-250.
21. HOLZINGER, A., TRAPNELL, B.C., WEAVER, T.E., WHITSETT, J.A., and IWAMOTO, H.S. (1995). Intraamniotic administration of an adenoviral vector for gene transfer to fetal sheep and mouse tissues. Pediatr. Res. 38, 844-850.
22. KELLY, E.J., and BROWNLEE, K.G. (1993). When is the fetus first capable of gastric acid, intrinsic factor and gastrin secretion? Biol. Neonate 63, 153-156.
23. KELLY, R.W., and NEWNHAM, J.P. (1989). Estimation of gestational age in Merino ewes by ultrasound measurement of fetal head size. Austr. J. Agric. Res. 40, 1293-1299.
24. LAU, C., SORIANO, H., and LEDLEY, F. (1995). Retroviral gene transfer into the intestinal epithelium. Hum. Gene Ther. 6, 1145-1151.
25. LE, L.P., EVERTS, M., DMITRIEV, I.P., DAVYDOVA, J.G., YAMAMOTO, M., and CURIEL, D.T. (2004). Fluorescently labeled adenovirus with pIX-EGFP for vector detection. Mol. Imaging 3, 105-116.
26. PEEBLES, D., GREGORY, L.G., DAVID, A., THEMIS, M., WADDINGTON, S., KNAPTON, H.J., MIAH, M., COOK, T., LAWRENCE, L., NIVSARKAR, M., RODECK, C., and COUTELLE, C. (2004). Widespread and efficient marker gene expression in the airway epithelia of fetal sheep after minimally invasive tracheal application of recombinant adenovirus in utero. Gene Ther. 11, 70-78.
27. PHILLIPS, A.D., and SCHMITZ, J. (1992). Familial microvillous atrophy: A clinicopathological survey of 23 cases. J. Pediatr. Gastroenterol. Nutr. 14, 380-396.
28. POLAK-CHARCON, S., SHOHAM, J., and BEN-SHAUL, Y. (1980). Tight junctions in epithelial cells of human fetal hindgut, normal colon, and colon adenocarcinoma. J. Natl. Cancer Inst. 65, 53-62.
29. PRETORIUS, D.H., GOSINK, B.B., CLAUTICE-ENGLE, T., LEOPOLD, G.R., and MINNICK, C.M. (1988). Sonographic evaluation of the fetal stomach: Significance of nonvisualization. Am. J. Roentgenol. 151, 987-989.
30. SAKAI, M., IMAI, T., OHTAKE, H., AZUMA, H., and OTAGIRI, M. (1997). Effects of absorption enhancers on the transport of model compounds in Caco-2 cell monolayers: Assessment by confocal laser scanning microscopy. J. Pharm. Sci. 86, 779-785.
31. SANDBERG, J., LAU, C., and JACOMINO, M. (1994). Improving access to intestinal stem cells as a step toward intestinal gene transfer. Hum. Gene Ther. 10, 13-18.
32. SHAO, G., BACKSTROM, K.C., HUANG, Q., and SFERRA, T. (2003). Gastric acid neutralization and protease inhibition improves oral recombinant adeno-associated virus mediated gene delivery to the intestines. Mol. Ther. 7, S389-S390.
33. SHULKES, A., CHICK, P., HARDY, K.J., ROBINSON, P., and TRAHAIR, J. (1985). Ontogeny of gastric acidity in the ovine fetus. J. Dev. Physiol. 7, 195-206.
34. SMITH, T., CHRISTIANSON, K., MOSS, R., and BAILEY, D. (1985). Structural and biochemical differentiation of the guinea-pig colon during foetal development. Cell Tissue Res. 242, 197-209.
35. STRATFORD-PERRICAUDET, L.D., MAKEH, I., PERRICAUDET, M., and BRIAND, P. (1992). Widespread long-term gene transfer to mouse skeletal muscles and heart. J. Clin. Invest. 90, 626-630.
36. STRONG, T.V., BOEHM, K., and COLLINS, F.S. (1994). Localization of cystic fibrosis transmembrane conductance regulator mRNA in the human gastrointestinal tract by in situ hybridization. J. Clin. Invest. 93, 347-354.
37. THEMIS, M., SCHNEIDER, H., KISERUD, T., COOK, T., ADEBAKIN, S., JEZZARD, S., FORBES, S., HANSON, M., PAVIRANI, A., RODECK, C., and COUTELLE, C. (1999). Successful expression of β-galactosidase and factor IX transgenes in fetal and neonatal sheep after ultrasound-guided percutaneous adenovirus vector administration into the umbilical vein. Gene Ther. 6, 1239-1248.
38. TREZISE, A.E., and BUCHWALD, M. (1991). In vivo cell-specific expression of the cystic fibrosis transmembrane conductance regulator. Nature 353, 434-437.
39. TREZISE, A.E., CHAMBERS, J.A., WARDLE, C.J., GOULD, S., and HARRIS, A. (1993). Expression of the cystic fibrosis gene in human foetal tissues. Hum. Mol. Genet. 2, 212-218.
40. TRIER, J.S., and MOXEY, P.C. (1979). Morphogenesis of the small intestine during fetal development. Ciba Found. Symp. 70, 3-29.
41. VERMA, K.B. (1979). Development of mucosa of the human ileum. J. Anat. 128, 512-521.
42. WEISS, D.J., STRANDJORD, T.P., JACKSON, J.C., CLARK, J.G., and LIGGITT, D. (1999). Perfluorochemical liquid-enhanced adenoviral vector distribution and expression in lungs of spontaneously breathing rodents. Exp. Lung Res. 25, 317-333.
43. WEISS, D.J., LIGGITT, H.D., and BONNEAU, L.A. (2000). Pretreatment with perfluorochemical liquid enhances adenoviral-mediated gene expression in lung. Pediatr. Pulmonol. Suppl. 20, 235.
44. WEISS, D.J., BONNEAU, L., and LIGGITT, D. (2001). Use of perfluorochemical liquid allows earlier detection of gene expression and use of less vector in normal lung and enhances gene expression in acutely injured lung. Mol. Ther. 3, 734-745.
45. WEISS, D.J., BASKIN, G.B., SHEAN, M.K., BLANCHARD, J.L., and KOLLS, J.K. (2002). Use of perflubron to enhance lung gene expression: Safety and initial efficacy studies in nonhuman primates. Mol. Ther. 5, 8.
46. WESTBROOK, C.A., CHMURA, S.J., ARENAS, R.B., KIM, S.Y., and OTTO, G. (1994). Human APC gene expression in rodent colonic epithelium in vivo using liposomal gene delivery. Hum. Mol. Genet. 3, 2005-2010.
47. WINTON, D.J., and PONDER, B.A. (1990). Stem-cell organization in mouse small intestine. Proc. Biol. Sci. 241, 13-18.
48. WU, Y., LIU, J., WOO, S., FINEGOLD, MJ., and BRANDT, M.L. (1999). Prenatal orogastric gene delivery results in transduction of the small bowel in the fetal rabbit. Fetal Diagn. Ther. 14, 323-327.