Ultrasound-guided gene transfer to hepatocytes in utero

Fetal Diagnosis and Therapy

Volumn 13, Issue 4, 1998, Pages 197-205

  Wang, Guoshun ^a   Williamson, Roger ^a   Mueller, Glenda ^a   Thomas, Patricia ^a   Davidson, Beverly L ^a   McCray Jr , Paul B ^a  

^a UNIVERSITY OF IOWA   (United States)

Author keywords

Animal model; Fetal gene therapy, rabbit; Gene therapy; Liver

Indexed keywords

BETA GALACTOSIDASE; VIRUS VECTOR;

ADENOVIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; FETUS; FETUS CIRCULATION; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; IMMUNE RESPONSE; LIVER CELL; NONHUMAN; PRIORITY JOURNAL; RABBIT; REPORTER GENE; ULTRASOUND;

ADENOVIRIDAE; ANIMALS; BETA-GALACTOSIDASE; FEMALE; FETAL BLOOD; GENE TARGETING; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; GESTATIONAL AGE; LIVER; LIVER DISEASES; PREGNANCY; RABBITS; ULTRASONICS;

EID: 0031736286     PISSN: 10153837     EISSN: None     Source Type: Journal    
DOI: 10.1159/000020838     Document Type: Article

References (59)

1. Chowdhury, JR, Wolkoff AW, Chowdhury NR, Arias IM: Hereditary jaundice and disorders of bilirubin metabolism; in Scriver CR, Beaudet AL, Sly WS, Valle D (eds): The Metabolic and Molecular Bases of Inherited Disease. New York, McGraw-Hill, 1995, pp 2161-2208.
2. Mitchell GA, Lambert M, Tanguay RM: Hypertyrosinemia; in Scriver CR, Beaudet AL, Sly WS, Valle D (eds). The Metabolic and Molecular Bases of Inherited Disease. New York, McGraw-Hill, 1995, pp 1077-1106.
3. Valle D, Simell O: The hyperornithinemias; in Scriver CR, Beaudet AL, Sly WS, Valle D (eds): The Metabolic and Molecular Bases of Inherited Disease. New York, McGraw-Hill, 1995, pp 1147-1185.
4. Grompe M, Jones SN, Loulseged H, Caskey CT: Retroviral-mediated gene transfer of human ornithine transcarbamylase into primary hepatocytes of spf and spf-ash mice. Hum Gene Ther 1992;3:35-44.
5. Branchereau PS, Ferry N, Myara A, Sato H, Kowai O, Trivin F, Houssin D, Danos O, Heard J: Correction of bilirubin glucuronyl-transferase deficiency in the Gunn rat with gene transfer into the liver via retrovirus vectors. Chirurgie 1994;119:642-648.
6. Ye X, Robinson MB, Pabin C, Quinn T, Jaward A, Wilson JM, Batshaw ML: Adenovirus-mediated in vivo gene transfer rapidly protects ornithine transcarbamylase-deficient mice from an ammonium challenge. Pediatr Res 1997;41:527-534.
7. Overtuf K, Al-Dhalimy M, Ou CN, Finegold M, Tanguay R, Lieber A, Kay M, Grompe M: Adenovirus-mediated gene therapy in a mouse model of hereditary tyrosinemia type I. Hum Gene Ther 1997;8:513-521.
8. Green RM, Gollan JL: Crigler-Najjar disease type I: Therapeutic approaches to genetic liver diseases into the next century. Gastroenterology 1997;112:649-651.
9. Askari F, Hitomi E, Thiney M, Wilson JM: Retrovirus-mediated expression of HUG Brl in Crigler-Najjar syndrome type I human fibroblasts and correction of the genetic defect in Gunn rat hepatocytes. Gene Ther 1995;2:203-208.
10. Askan FK, Hitomi Y, Mao M, Wilson JM: Complete correction of hyperbilirubinemia in the Gunn rat model of Crigler-Najjar syndrome type I following transient in vivo adenovirus-mediated expression of human bilirubin UDP-glucuronosyltransferase. Gene Ther 1996;3:381-388.
11. Ledley FD: Prenatal application of somatic gene therapy. Obstet Gynecol Clin North Am 1993;20:611-621.
12. McCray PB Jr: In-utero gene transfer - an approach to the treatment of inherited lung diseases. Mol Hum Reprod 1996;2:469-471.
13. Coutelle C, Douar A, Colledge WH, Froster U: The challenge of fetal gene therapy. Nat Med 1995;1:864-866.
14. Kohn DB, Parkman R: Gene therapy for newborns. FASEB J 1997;11:635-639.
15. Evans MI, Harrison MR, Nicolaides KH, Johnson MP, Holzgreve W: Fetal therapy; in Milunsky A (ed): Genetic Disorders and the Fetus: Diagnosis, Prevention, Treatment. Baltimore, Johns Hopkins University Press, 1992, pp 771-797.
16. Adzick NS, Harrison MR: Fetal surgical therapy. Lancet 1994;343:897-902.
17. Ciotti M, Obaray R, Martin MG, Owens IS: Genetic defects at the UGT1 locus associated with Crigler-Najjar type I disease, including a prenatal diagnosis. Am J Med Genet 1997;68:173-178.
18. Wilhamson RA, Varner MW, Grant SS: Reduction in amniocentesis risks using a real-time needle guide procedure. Obstet Gynecol 1985;65:751-755.
19. Reece RA, Homko CJ, Koch S, Chan L: First-trimester needle embryofetoscopy and prenatal diagnosis. Fetal Diagn Ther 1997;12:136-139.
20. Reece EA, Homko C, Goldstein I, Wiznitzer A: Toward fetal therapy using needle embryofetoscopy. Ultrasound Obstet Gynecol 1995;5:281-285.
21. Engelhardt JF, Ye X, Doranz B, Wilson JM: Ablation of E2A in recombinant adenoviruses improves transgene persistence and decreases inflammatory response in mouse liver. Proc Natl Acad Sci USA 1994;91:6196-6200.
22. Jaffe HA, Danel C, Longenecker G, Metzger M, Setoguchi Y, Rosenfeld MA, Gant TW, Thorgeirsson SS, Stratford-Perricaudet LD, Perricaudet M, Pavirani A, Lecocq JP, Crystal RG: Adenovirus-mediated in vivo gene transfer and expression in normal rat liver. Nat Genet 1992;1:372-378.
23. Bosch A, McCray PB Jr, Chang SM, Ulich TM, Simonet WS, Jolly DJ, Davidson EL: Proliferation induced by keratinocyte growth factor enhances in vivo retroviral-mediated gene transfer to mouse hepatocytes. J Clin Invest 1996;98:2683-2687.
24. Huard J, Lochmuller H, Acsadi G, Jani A, Massie B, Karpati G: The route of administration is a major determinant of the transduction efficiency of rat tissues by adenoviral recombinants. Gene Ther 1995;2:107-115.
25. Yang Y, Nunes FA, Berencsi K, Funh EE, Gonczol E, Wilson JM: Cellular immunity to viral antigens limits El-deleted adenoviruses for gene therapy. Proc Natl Acad Sci USA 1994;91:4407-4411.
26. Yang Y, Ertl HCJ, Wilson JM: MHC class I-restricted cytotoxic T lymphocytes to viral antigens destroy hepatocytes in mice infected with El-deleted recombinant adenoviruses. Immunity 1994;1:433-442.
27. Stratford-Perricaudet LD, Makeh I, Perricaudet M, Briand P: Widespread long-term gene transfer to mouse skeletal muscles and heart. J Clin Invest 1992;90:626-630.
28. Sekhon HS, Larson JE: In utero gene transfer into the pulmonary epithelium. Nat Med 1995;1:1201-1203.
29. Douar A-M, Adebakin S, Themis M, Pavirani A, Cook T, Coutelle C: Foetal gene delivery in mice by intraamniotic administration of retroviral producer cells and adenovirus. Gene Ther 1997;4:883-890.
30. Zepeda M, Wilson JM: Neonatal cotton rats do not exhibit destructive immune responses to adenoviral vectors. Gene Ther 1996;3:973-979.
31. Kass-Eisler A, Falck-Pedersen E, Elfenbein DH, Alvira M, Buttrick PM, Leinwand LA: The impact of developmental stage, route of administration and the immune system on adenovirus-mediated gene. Gene Ther 1994;1:395-402.
32. Davidson BL, Allen ED, Kozarsky KF, Wilson JM, Roessler BJ: A model system for in vivo gene transfer into the central nervous system using an adenoviral vector. Nat Genet 1993;3:219-223.
33. Moise KJ Jr, Hesketh DE, Belfort MM, Saade G, Van den Veyer IB, Hudson KM, Rodkey LS: Ultrasound-guided blood sampling of rabbit fetuses. Lab Anim Sci 1992;42:398-401.
34. McCray PB Jr, Armstrong K, Zabner J, Miller DW, Koretzky GA, Couture L, Robillard JE, Smith AE, Welsh MJ: Adenoviral-mediated gene transfer to fetal pulmonary epithelia in vitro and in vivo. J Clin Invest 1995;95:2620-2632.
35. Yang Y, Trinchieri G, Wilson JM: Recombinant IL-12 prevents formation of blocking IgA antibodies to recombinant adenovirus and allows repeated gene therapy to mouse lung. Nat Med 1995;1:890-893.
36. Zsengeller ZK, Wert SE, Hull WM, Hu X, Yei S, Trapnell BC, Whitsett JA: Persistence of replication-deficient adenovirus-mediated gene transfer in lungs of immune-deficient (nu/nu) mice. Hum Gene Ther 1995;6:457-467.
37. Vincent MC, Trapnell BC, Baughman RP, Wen SE, Whitsett JA, Iwamoto HS: Adenovirus-mediated gene transfer to the respiratory tract of fetal sheep in utero. Hum Gene Ther 1995;6:1019-1028.
38. Miyasaka M, Morris B: The ontogeny of the lymphoid system and immune responsiveness in sheep. Prog Vet Microbiol Immunol 1988;4:21-55.
39. Fahey KJ, Morris B: Humoral immune responses in foetal sheep. Immunology 1978;35:651-661.
40. Flake AW, Roncarolo M, Puck JM, Almeida-Porada G, Evans MI, Johnson MP, Abella EM, Hamson DD, Zanjani ED: Treatment of X-linked severe combined immunodeficiency by in utero transplantation of paternal bone marrow. N Engl J Med 1996;335:1806-1810.
41. Werner CP, Wenstrom KD, Sipes SL, Williamson RA: Risk factors for cordocentesis and fetal intravascular transfusion. Am J Obstet Gynecol 1991;165:1020-1025.
42. Holzinger A, Trapnell BC, Weaver TE, Whitsett JA, Iwamoto HS: Intraamniotic administration of an adenoviral vector for gene transfer to fetal sheep and mouse tissues. Pediatr Res 1995;38:844-850.
43. Hatzoglou M, tamers W, Bosch F, Wynshaw-Boris A, Clapp DW, Hanson RW: Hepatic gene transfer in animals using retroviruses containing the promoter from the gene for phosphoenolpyruvate carboxykinase. J Biol Chem 1990;265:17285-17293.
44. Hatzoglou M, Moorman A, Lamers W: Persistent expression of genes transferred in the fetal rat liver via retroviruses. Somat Cell Mol Genet 1995;21:265-278.
45. Pitt BR, Schwarz MA, Pilewski JM, Nakayama D, Mueller GM, Robbins PD, Watkins SA, Albertine KH, Bland RD: Retro virus-mediated gene transfer in lungs of living fetal sheep. Gene Ther 1995;2:344-350.
46. Tsukamoto M, Ochiya T, Yoshida S, Sugimura T, Terada M: Gene transfer and expression in progeny after intravenous DNA injection into pregnant mice. Nat Genet 1995;9:243-248.
47. Clapp DW, Dumenco LL, Hatzoglou M, Gerson SL: Fetal liver hematopoietic stem cells as a target for m utero retroviral gene transfer. Blood 1991;78:1132-1139.
48. Kozarsky KF, McKinley DR, Austin LL, Raper SE, Stratford-Perricaudet LD, Wilson JM: In vivo correction of low density lipoprotein receptor deficiency in the Watanabe heritable hyperhpidemic rabbit with recombinant adenoviruses. J Biol Chem 1994;269:13695-13702.
49. Gao GP, Yang Y, Wilson JM: Biology of adenovirus vectors with El and E4 deletions for liver-directed gene therapy. J Virol 1996;70:8934-8943.
50. Gorziglia MI, Kadan MJ, Yei S, Lim J, Lee GM, Luthra R, Trapnell BC: Elimination of both E1 and E2a from adenovirus vectors further improves prospects for in vivo human gene therapy. J Virol 1996;70:4173-4178.
51. Lieber A, He CY, Kirillova I, Kay MA: Recombinant adenoviruses with large deletions generated by cre-mediated excision exhibit different biological properties compared with first-generation vectors in vitro and in vivo. J Virol 1996;70:8944-8960.
52. Mitani K, Graham FL, Caskey CT, Kochanek S: Rescue, propagation, and partial purification of a helper virus-dependent adenovirus vector. Proc Natl Acad Sci USA 1995;92:3854-3858.
53. Kay MA, Holterman AX, Meuse L, Gown A, Ochs HD, Linsley PS, Wilson CB: Long-term hepatic adenovirus-mediated gene expression in mice following CTLA4Ig administration. Nat Genet 1995;11:191-197.
54. Kaplan JM, Smith AE: Transient immunosuppression with deoxyspergualin improves longevity of transgene expression and ability to readminister adenoviral vector to the mouse lung. Hum Gene Ther 1997;8:1095-1104.
55. Kessler PD, Podsakoff GM, Chen X, McQuiston SA, Colosi PC, Matelis LA, Kurtzman GJ, Byme BJ: Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc Natl Acad Sci USA 1995;93:14082-14087.
56. Koeberl DD, Alexander IE, Halben CL, Russell DW, Miller AD: Persistent expression of human clotting factor IX from mouse liver after intravenous injection of adeno-associated virus vectors. Proc Natl Acad Sci USA 1997;94:1426-1431.
57. Moullier P, Bohl D, Heard J-M, Danos O: Correction of lysosomal storage in the liver and spleen of MPS VII mice by implantation of genetically modified skin fibroblasts. Nat Genet 1993:4:154-159.
58. 1-antitrypsin in dogs after autologous transplantation of retroviral transduced hepatocytes. Proc Natl Acad Sci USA 1992;89:89-93.
59. Weiner CP, Williamson RA, Wenstrom KD, Sipes SL, Grant SS, Widness JA: Management of fetal hemolytic disease by cordocentesis. 1. Prediction of fetal anemia. Obstet Gynecol 1991;165:546-553.