Perspectives series: Molecular medicine in genetically engineered animals. Animal models of human disease for gene therapy

Journal of Clinical Investigation

Volumn 97, Issue 5, 1996, Pages 1138-1141

  Wilson, James M ^a,b  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b WISTAR INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOSINE DEAMINASE; BETA GLUCURONIDASE; GLUCOSYLCERAMIDASE; HYPOXANTHINE PHOSPHORIBOSYLTRANSFERASE; LOW DENSITY LIPOPROTEIN RECEPTOR; ORNITHINE CARBAMOYLTRANSFERASE; TRANSMEMBRANE CONDUCTANCE REGULATOR;

ANIMAL EXPERIMENT; ANIMAL MODEL; COMBINED IMMUNODEFICIENCY; CYSTIC FIBROSIS; ENZYME DEFICIENCY; EXPRESSION VECTOR; FAMILIAL HYPERCHOLESTEROLEMIA; GAUCHER DISEASE; GENE MUTATION; GENE THERAPY; GENETIC ENGINEERING; HYPERAMMONEMIA; LESCH NYHAN SYNDROME; MOUSE; MUCOPOLYSACCHARIDOSIS TYPE 7; NONHUMAN; PRIORITY JOURNAL; REVIEW; SCID MOUSE; TRANSGENIC MOUSE;

ANIMALS; DISEASE MODELS, ANIMAL; GENE THERAPY; GENETIC ENGINEERING; HUMANS;

EID: 0030003590     PISSN: 00219738     EISSN: None     Source Type: Journal    
DOI: 10.1172/jci118527     Document Type: Review

References (31)

1. United States Department of Health and Human Services/NIH. 1991. Proposed actions under the guidelines and advisory committee meeting: Notice. In Recombinant DNA Research. Vol. 14. 599-603.
2. Anderson, W.F. 1984. Prospects for Human Gene Therapy. Science (Wash. DC). 226:401-409.
3. Seegmiller, J.E., F.M. Rosenbloom, and W.N. Kelley 1967. An enzyme defect associated with a sex-linked human neurological disorder and excessive purine synthesis. Science (Wash. DC). 155:1682-1684
4. Stout, J.T., and C.T. Caskey. 1989. Hypoxanthine phosphoribosyltransferase deficiency: the Lesch-Nyhan syndrome and gouty arthritis. In The Metabolic Basis of Inherited Disease. Vol. 1. C.R. Scriver, A L. Beaudet, W.S. Sly, and D. Valle, editors. McGraw-Hill, Inc., New York. 1007-1028.
5. Hooper, M., K Hardy, A. Handyside, S. Hunter, and M. Monk. 1987. HPRT deficient (Lesch-Nyhan) mouse embryos derived from germline colonisation by cultured cells. Nature (Lond.). 326:292-295.
6. Kuehn, M.R., A. Bradley, E.J. Robertson, and M.J. Evans. 1987. A potential animal model for Lesch-Nyhan syndrome through introduction of HPRT mutations into mice. Nature (Lond.). 326:295-298.
7. Giblett, E.R., J.E. Anderson, F. Cohen, B. Pollara, and H.J. Meuwissen 1972. Adenosine deaminase deficiency in two patients with severely impaired cellular immunity. Lancet. ii:1067-1069.
8. Anderson, W.F., R.M. Blaese, and K. Culver. 1990. The ADA human gene therapy clinical protocol. Hum. Gene Ther. 1:327-362.
9. Blaese, M.R., K.W. Culver, A.D. Miller, C.S. Carter, T. Fleisher, M. Clerici, G. Shearer, L. Chang, Y. Chiang, P. Tolstoshev, et al. 1995. T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years. Science (Wash. DC). 270:475-480.
10. Kohn, D.B., K.I. Weinberg, J.A. Nolta, L.N. Heiss, C. Lenarsky, G.M. Crooks, M E. Hanley, G. Annett, J.S. Brooks, A. El-Khoureiy, et al. 1995. Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency. Nature Medicine 1:1017-1023
11. Migchielsen, A.A.J., M.L Breuer, M.A van Roon, H. te Riele, C. Zurcher, F. Ossendorp, S. Toutain, M.S. Hershfield, A. Berns, and D. Valerio. 1995. Adenosine-deaminase-deficient mice die perinatally and exhibit liver-cell degeneration, atelectasis and small intestinal cell death. Nat. Genet. 10:279-287.
12. Wolfe, J.H., M.S. Sands, J.E. Barker, B. Gwynn, L.B. Rowe, C.A. Vogler, and E.H. Birkenmeier. 1992. Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer. Nature (Lond). 360: 749-753.
13. Stratford-Perricaudet, L.D., M. Levrero, J.F. Chasse, M. Perricaudet, and P. Briand. 1990. Evaluation of the transfer and expression in mice of an enzyme-encoding gene using a human adenovirus vector. Hum. Gene Ther. 1:241-256.
14. Ishibashi, S., M. Brown, J. Goldstein, R. Gerard, R Hammer, and J. Herz. 1993. Hypercholesterolemia in low density lipoprotein receptor knockout mice and its reversal by adenovirus-mediated gene delivery. J. Clin. Invest. 92: 883-893.
15. Tybulewicz, V.L.J., M.L. Tremblay, M.E. LaMarca, R. Willemsen, B.K. Stubblefield, S. Winfield, B. Zablocka, E. Sidransky, B.M. Martin, S.P. Huang, et al. 1992. Animal model of Gaucher's disease from targeted disruption of the mouse glucocerebrosidase gene Nature (Lond.). 357:407-410.
16. Snouwaert, J.N., K.K. Brigman, A.M. Latour, N.N. Malouf, R.C. Boucher, O. Smithies, and B.H Koller. 1992. An animal model for cystic fibrosis made by gene targeting Science (Wash. DC). 257:1083-1088.
17. Dorin, J.R., P. Dickinson, E.W.F.W. Alton, S.N. Smith, D.M. Geddes, B.J. Stevenson, W.L. Kimber, S. Fleming, A.R. Clarke, M.L. Hooper, et al. 1992. Cystic fibrosis in the mouse by targeted insertional mutagenesis. Nature (Lond.) 359:211-215.
18. Colledge, W.H., B.S. Abella, K.W. Southern, R. Ratcliff, C. Jiang, S.H. Cheng, L.J. MacVinish, J.R. Anderson, A.W. Cuthbert, and M.J. Evans. 1995. Generation and characterization of a ΔF508 cystic fibrosis mouse model. Nat. Genet. 10:445-452.
19. Zhou, L., C.R. Dey, S.E Wert, M.D. DuVall, R.A. Frizzell, and J.A. Whitsett. 1994. Correction of lethal intestinal defect in a mouse model of cystic fibrosis by human CFTR. Science (Wash. DC). 266:1705-1708.
20. Davidson, D., J. Dorin, G. McLachlan, V. Ranald, D. Lamb, C. Doherty, J. Govan, and D. Porteous 1995. Lung disease in the cystic fibrosis mouse exposed to bacterial pathogens. Nat. Genet 9:351-357.
21. Clarke, L.L., B.R. Grubb, J.R. Yankaskas, C.U Cotton, A. McKenzie, and R.C Boucher 1994. Relationship of a non-cystic fibrosis transmembrane conductance regulator-mediated chloride conductance to organ-level disease in CFTR (-/-) mice. Proc. Natl. Acad. Sci. USA. 91:479-483.
22. Wilson, J.M. 1995. Gene therapy for cystic fibrosis, challenges and future directions. J. Clin. Invest. 96:2547-2554.
23. Yang, Y., H. Ertl, and J. Wilson. 1994. MHC class 1 restricted cytotoxic T lymphocytes to viral antigens destroy hepatocytes in mice infected with E1 deleted recombinant adenoviruses. Immunity. 1:433-442.
24. Yang, Y., Q. Li, H Ertl, and J. Wilson. 1995. Cellular and humoral immune responses to viral antigens create barriers to lung-directed gene therapy with recombinant adenoviruses. J Virol. 69:2004-2015.
25. Dai, Y., E. Schwartz, D. Gu, W. Zhang, N. Sarvetnick, and I. Verma. 1995. Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: tolerization of factor IX and vector antigens allows for long-term expression. Proc. Natl Acad. Sci. USA. 92:1401-1405.
26. Chowdhury, J., M. Grossman, S. Gupta, N. Chowdhury, J. Baker, and J. Wilson. 1991. Long-term improvement of hypercholesterolemia after ex vivo gene therapy in LDLR deficient rabbits. Science (Wash. DC). 254:1802-1805.
27. Grossman, M., S. Raper, K. Kozarsky, E. Stein, J. Engelhardt, D. Muller, and J. Wilson. 1994. Successful ex vivo gene therapy directed to liver patient with familial hypercholesterolemia. Nat Genet. 6:335-341.
28. Grossman, M., D J. Rader, D.W.M. Muller, D.M. Kolansky, K. Kozarsky, B.J. Clark, E.A. Stein, P.J. Lupien, H.B. Brewer, S.E. Raper, and J.M. Wilson. 1995. A pilot study of ex vivo gene therapy for homozygous familial hypercholesterolemia. Nature Medicine. 1:1148-1154.
29. Rosenfeld, M.A., K. Yoshimura, B.C. Trapnell, K Yoneyama, E.R. Rosenthal, W. Dalemans, M. Fukayama, J. Bargon, L.E. Stier, L. Stratford-Perricaudet, et al. 1992. In vivo transfer of the human cystic fibrosis transmembrane conductance regulator gene to the airway epithelium. Cell. 68:143-155.
30. Yang, Y., F. Nunes, K. Berencsi, E. Gønczøl, J. Engelhardt, and J. Wilson. 1994. Inactivation of E2a in recombinant adenoviruses improves the prospect for gene therapy in cystic fibrosis. Nat. Genet. 7:362-369.
31. Crystal, R.G., N.G. McElvaney, M.A. Rosenfeld, C.-S. Chu, A. Mastrangeli, J.G. Hay, S.L. Brody, H.A. Jaffe, N.T. Eissa, and C Danel. 1994. Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis. Nat. Genet. 8:42-51.