Gene transfer strategies for metabolic diseases

World Journal of Surgery

Volumn 26, Issue 7, 2002, Pages 838-842

  Raper, Steven E ^a   McClane, Steven J ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

VIRUS VECTOR;

BIOTECHNOLOGY; CONFERENCE PAPER; FAMILIAL HYPERCHOLESTEROLEMIA; GENE TRANSFER; LIVER; MEDICAL PRACTICE; METABOLIC DISORDER; MOLECULAR GENETICS; PANCREAS; PATIENT CARE; SURGERY; TECHNOLOGY; ADENOVIRUS; ARTICLE; GENE THERAPY; GENE VECTOR; GENETICS; HUMAN; HYPERLIPOPROTEINEMIA TYPE 2; IMMUNOHISTOCHEMISTRY; METHODOLOGY; PROTO ONCOGENE;

ADENOVIRIDAE; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; HYPERLIPOPROTEINEMIA TYPE II; IMMUNOHISTOCHEMISTRY; PROTO-ONCOGENES;

EID: 0036631850     PISSN: 03642313     EISSN: None     Source Type: Journal    
DOI: 10.1007/s00268-002-4061-5     Document Type: Conference Paper

References (28)

1. Kozarsky KF, McKinley DR, Austin LL, et al. In vivo correction of low density lipoprotein receptor deficiency in the Watanabe heritable hyperlipidemic rabbit with recombinant adenoviruses. J. Biol. Chem. 1994;269:13695
2. Tsukamoto K, Smith P, Glick J, et al. Liver-directed gene transfer and prolonged expression of three major human apo-E isoforms in apo-E deficient mice. J. Clin. Invest. 1997;100:107
3. Raper SE. The four-legged hepatocyte: lessons learned about cholesterol metabolism from new technologies. Hepatology 1996;24:463
4. Ye X, Robinson MB, Batshaw ML, et al. Prolonged metabolic correction in adult ornithine transcarbamylase-deficient mice with adenoviral vectors. J. Biol. Chem. 1996;271:3639
5. Kolodka TM, Finegold M, Moss L, et al. Gene therapy for diabetes mellitus in rats by hepatic expression of insulin. Proc. Natl. Acad. Sci. U.S.A. 1995;92:3293
6. Wilson JM. Adenoviruses as gene-delivery vehicles. N. Engl. J. Med. 1996;334:1185
7. Raper SE, Haskal ZJ, Ye X, et al. Selective gene transfer into the liver of non-human primates with E1-deleted, E2A-defective, or E1-E4 deleted recombinant adenoviruses. Hum. Gene Ther. 1998;9:671
8. Sterman DH, Treat J, Litzky LA, et al. Adenovirus-mediated herpes simplex virus thymidine kinase/ganciclovir gene therapy in patients with localized malignancy: results of a phase I clinical trial in malignant mesothelioma. Hum. Gene Ther. 1998;9:1083
9. Smith JG, Raper SE, Wheeldon EB, et al. Intracranial administration of adenovirus expressing HSV-TK in combination with ganciclovir produces a dose-dependent, self-limiting inflammatory response. Hum. Gene Ther. 1997;8:943
10. Yang Y, Nunes FA, Berencsi K, et al. Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy. Proc. Natl. Acad. Sci. U.S.A. America 1994;91:4407
11. Yang Y, Ertl HC, Wilson JM. MHC class I-restricted cytotoxic T lymphocytes to viral antigens destroy hepatocytes in mice infected with E1-deleted recombinant adenoviruses. Immunity 1994;1:433
12. Yang Y, Wilson JM. CD40 ligand-dependent T cell activation: requirement of B7-CD28 signaling through CD40. Science 1996;273:1862
13. Jooss K, Ertl HC, Wilson JM. Cytotoxic T-lymphocyte target proteins and their major histocompatibility complex class I restriction in response to adenovirus vectors delivered to mouse liver. J. Virol. 1998;72:2945
14. Gao GP, Yang Y, Wilson JM. Biology of adenovirus vectors with E1 and E4 deletions for liver-directed gene therapy. J. Virol. 1996;70:8934
15. Fisher KJ, Choi H, Burda J, et al. Recombinant adenovirus deleted of all viral genes for gene therapy of cystic fibrosis. Virology 1996;217:11
16. Morsy M, Gu M, Motzel S, et al. An adenoviral vector deleted for all viral coding sequences results in enhanced safety and extended expression of a leptin transgene. Proc. Natl. Acad. Sci. U.S.A. 1998;95:7866
17. Sadowski H, Shuai K, Darnell JJ, et al. A common nuclear signal transduction pathway activated by growth factor and cytokine receptors. Science 1993;261:1739
18. Shuai K, Stark G, Kerr I, et al. A single phosphotyrosine residue of Stat91 required for gene activation by interferon-γ. Science 1993;261:1744
19. Larner A, David M, Feldman G, et al. Tyrosine phosphorylation of DNA binding proteins by multiple cytokines. Science 1993;261:1730
20. Zhong Z, Wen Z, Darnell JJ. STAT3: a STAT family member activated by tyrosine phosphorylation in response to epidermal growth factor and interleukin-6. Science 1994;264:95
21. Schlossberg H, Zhang Y, Dudus L, et al. Expression of c-fos and c-jun during hepatocellular remodelling following ischemia/reperfusion in mouse liver. Hepatology 1996;23:1546
22. Haber B, Mohn K, Diamond R, et al. Induction patterns of 70 genes during nine days after hepatectomy define the temporal course of liver regeneration. J. Clin. Invest. 1993;91:1319
23. McClane SJ, Chirmule N, Burke CV, et al. Characterization of the immune response after local delivery of recombinant adenovirus in murine pancreas and successful strategies for readministration. Hum. Gene Ther. 1997;8:2207
24. McClane SJ, Hamilton TE, DeMatteo RP, et al. Effect of adenoviral early genes and the host immune system on in vivo pancreatic gene transfer in the mouse. Pancreas 1997;15:236
25. Grossman M, Raper SE, Wilson JM. Transplantation of genetically modified autologous hepatocytes into nonhuman primates: feasibility and short-term toxicity. Hum. Gene Ther. 1992;3:501
26. Grossman M, Wilson JM, Raper SE. A novel approach for introducing hepatocytes into the portal circulation. J. Lab. Clin. Med. 1993;121:472
27. Raper SE, Grossman M, Rader DJ, et al. Safety and feasibility of liver-directed ex vivo gene therapy for homozygous familial hypercholesterolemia. Ann. Surg. 1996;223:116
28. Grossman M, Rader DJ, Muller DW, et al. A pilot study of ex vivo gene therapy for homozygous familial hypercholesterolaemia. Nat. Med. 1995;1:1148