Progress and Potential for Gene-Based Medicines

Molecular Therapy

Volumn 1, Issue 3, 2000, Pages 213-224

  Dyer, Mark R ^a   Herrling, Paul L ^a  

^a NOVARTIS PHARMA AG   (Switzerland)

Author keywords

[No Author keywords available]

Indexed keywords

VIRUS DNA; VIRUS RNA;

ANIMAL; BIOETHICS; BIOSYNTHESIS; CLINICAL TRIAL; GENE EXPRESSION REGULATION; GENE THERAPY; GENE TRANSFER; GENE VECTOR; GENETICS; HUMAN; METHODOLOGY; ORGAN TRANSPLANTATION; REVIEW; XENOGRAFT;

ANIMALS; BIOETHICS; CLINICAL TRIALS; DNA, VIRAL; GENE EXPRESSION REGULATION; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; ORGAN TRANSPLANTATION; RNA, VIRAL; TRANSPLANTATION, HETEROLOGOUS;

EID: 0034152565     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1006/mthe.2000.0044     Document Type: Review

References (37)

1. Cohen D., Chumakov I., Weissenbach J. A first-generation physical map of the human genome. Nature. 366:1993;698-701.
2. Watson J. D. The human genome project: Past, present and future. Science. 248:1990;44-51.
3. Fields C., Adams M. D., White O., Venter J. C. How many genes in the human genome. Nature Genet. 7:1994;345-346.
4. Dunham I. The DNA sequence of human chromosome 22. Nature. 402:1999;489-495.
5. Selkoe D. J. Alzheimer's disease: Genotypes, phenotypes and treatments. Science. 275:1997;630-631.
6. Goedert M. Familial Parkinson's disease. The awakening of alpha-synuclein. Nature. 388:1997;232-233.
7. Polymeropoulos M. H., Lavedan C., Leroy E., Ide S. E., Dehejia A., Dutra A., Pike B., Root H., Rubenstein J., Boyer R., Stenroos E. S., Chandrasekharappa S., Athanassiadou A., Papapetropoulos T., Johnson W. G., Lazzarini A. M., Duvoisin R. C., Di-Iorio G., Golbe L. I., Nussbaum R. L. Mutation in the alpha-synuclein gene identified in families with Parkinson's disease. Science. 276:1997;2045-2047.
8. Cordell H. J., Todd J. A. Multifactorial inheritance in type I diabetes. Trends Genet. 11:1995;499-504.
9. Postma D. S., Bleecker E. R., Amelung P. J., Holroyd K. J., Xu J., Panhuysen C. I. M., Meyers D. A., Levitt R. C. Genetic susceptibility to asthma - Bronchial hyperresponsiveness coinherited with a major gene for atopy. N. Engl. J. Med. 333:1995;894-900.
10. Maddison P. J., Isenberg D. A., Woo P., Glass D. N. Oxford Textbook of Rheumatology. 1993;Oxford Medical Publications.
11. Druker J. B., Tamura S., Buchdunger E., Ohno S., Segal G. M., Fanning S., Zimmermann J., Lydon N. B. Effects of a selective inhibitor of the Abl tyrosine kinase on the growth of Bcr-Abl positive cells. Nat. Med. 2:1996;561-566.
12. Dyer M. R., Cohen D., Herrling P. L. Functional genomics: From genes to new therapies. Drug Discovery Today. 4:1999;109-114.
13. Anderson W. F. Human gene therapy. Nature. 392:1998;25-30.
14. Anderson W. F. Prospects for human gene therapy. Science. 226:1984;401-409.
15. Amado R. G., Chen I. S. Lentiviral vectors - The promise of gene therapy within reach. Science. 285:1999;674-676.
16. Trono D. Lentiviral vectors: Turning a deadly foe into a therapeutic agent. Gene Ther. 7:2000;20-23.
17. Ali M., Lemoine N. R., Ring J. A. The use of DNA viruses as vectors for gene therapy. Gene Ther. 1:1994;367-384.
18. Monahan P. E., Samulski R. J. AAV vectors: Is clinical success on the horizon. Gene Ther. 7:2000;24-30.
19. Langer R. Drug delivery and targeting. Nature. 392:1998;5-10.
20. Li S., Huang L. Nonviral gene therapy: Promises and challenges. Gene Ther. 7:2000;31-34.
21. Kaneda Y. Development of a novel fusogenic viral liposome system (HVJ-liposomes) and its applications to the treatment of acquired diseases. Mol. Membr. Biol. 16:1999;119-122.
22. Alton E. W., Stern M., Farley R., Jaffe A., Chadwick S. L., Phillips J., Davies J., Smith S. N., Browning J., Davies M. G., Hodson M. E., Durham S. R., Li D., Jeffery P. K., Scallan M., Balfour R., Eastman S. J., Cheng S. H., Smith A. E., Meeker D., Geddes D. M. Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: A double-blind placebo-controlled trial. Lancet. 353:1999;947-954.
23. Gage F. H. Cell therapy. Nature. 392:1998;18-24.
24. Su L., Lee R., Bonyhadi M., Matsuzaki H., Forestell S., Escaich S., Bohnlein E., Kaneshima H. Hematopoietic stem cell-based gene therapy for acquired immunodeficiency syndrome: Efficient transduction and expression of RevM10 in myeloid cells in vivo and in vitro. Blood. 89:1997;2283-2290.
25. Baron U., Gossen M., Bujard H. Tetracycline-controlled transcription in eukaryotes: Novel transactivators with graded transactivation potential. Nucleic Acids Res. 25:1997;2723-2729.
26. Pollock R., Rivera V. M. Regulation of gene expression with synthetic dimerizers. Methods Enzymol. 306:1999;263-281.
27. http://www4.od.nih.gov/oba/documents.htm .
28. Hollon T. Researchers and regulators reflect on first gene therapy death. Nat. Med. 6:2000;6.
29. Dewey R. A., Morrissey G., Cowsill C. M., Stone D., Bolognani F., Dodd N. J. F., Southgate T. D., Klatzmann D., Lassmann H., Castro M. G., Löwenstein P. R. Chronic brain inflammation and persistent herpes simplex virus 1 thymidine kinase expression in survivors of syngeneic glioma treated by adenovirus-mediated gene therapy: Implications for clinical trials. Nat. Med. 5:1999;1256-1263.
30. Auchincloss H., Sachs D. H. Xenogenic transplantation. Annu. Rev. Immunol. 16:1998;433-470.
31. Guidance for Industry: Public Health Issues Posed by the Use of Nonhuman Primate Xenografts in Humans, http://www.fda.gov/cber/gdlns/xenoprim.txt .
32. Bhatti F. N., Schmoeckel M., Zaidi A., Cozzi E., Chavez G., Goddard M., Dunning J. J., Wallwork J., White D. J. Three-month survival of HDAF transgenic pig hearts transplanted into primates. Transplant. Proc. 31:1999;958.
33. Paradis K., Langford G., Long Z., Heneine W., Sandstrom P., Switzer W. M., Chapman L. E., Lockey C., Onions D., Otto E. Search for cross-species transmission of porcine endogenous retrovirus in patients treated with living pig tissue. Science. 285:1999;1236-1241.
34. Birmingham K. FDA subcommittee finds no evidence of PERV transmission. Nat. Med. 5:1999;855.
35. Wilson C., Ng T. H., Miller A. E. Evaluation and recommendations for replication-competent retrovirus testing associated with use of retroviral vectors. Hum. Gene Ther. 8:1997;869-874.
36. Long Z., Lu P., Grooms T., Mychkovsky I., Westley T., Fitzgerald T., Sharma-Chibber S., Shand N., McGarrity G., Otto E. Molecular evaluation of biopsy and autopsy specimens from patients receiving in vivo retroviral gene therapy. Hum. Gene Ther. 10:1999;733-740.
37. Knoppers B. M., Hirtle M., Lormeau S., Laberge C. M., Laflamme M. Control of DNA samples and information. Genomics. 50:1998;385-401.