Vectors and target cells for gene therapy of blood diseases

Trends in Cardiovascular Medicine

Volumn 6, Issue 1, 1996, Pages 25-30

  Qazilbash, Muzaffar ^a   Young, Neal ^a   Liu, Johnson ^a  

^a NATIONAL CANCER INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOSINE DEAMINASE; LIPOSOME;

ACQUIRED IMMUNE DEFICIENCY SYNDROME; BLOOD DISEASE; COMBINED IMMUNODEFICIENCY; GENE TARGETING; GENE THERAPY; GENE TRANSFER; GENETIC DISORDER; GENETIC MARKER; HEMATOPOIETIC STEM CELL; IMMUNOMODULATION; PRIORITY JOURNAL; RETROVIRUS; SHORT SURVEY;

EID: 0030025194     PISSN: 10501738     EISSN: None     Source Type: Journal    
DOI: 10.1016/1050-1738(95)00127-1     Document Type: Short Survey

References (37)

1. Ascadi G, Dickson G, Love DR, et al.: 1991. Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs. Nature 352:815-818.
2. Blaese RM: 1993. Development of gene therapy for immunodeficiency: adenosine deaminase deficiency. Pediatr Res 33(Suppl): S49-S55.
3. - SCID: initial trial results after 4 years. Science 270:475-480.
4. Brenner MK, Rill DR, Moen RC, et al.: 1993. Gene marking to trace origin of relapse after autologous bone marrow transplantation. Lancet 341:85-86.
5. Cheng L, Ziegelhofffer PR, Yang NS: 1993. In vivo promoter activity and transgene expression in mammalian somatic tissues evaluate by using particle bombardment. Proc Natl Acad Sci USA 90:4455-4459.
6. Culver K, Cornetta K, Morgan R, et al.: 1991. Lymphocytes as cellular vehicles for gene therapy in mouse and man. Proc Natl Acad Sci USA 88:3155-3159.
7. Culver KW, Ram Z, Walbridge S, et al.: 1992. In vivo gene transfer with retroviral vector producer cells for treatment of experimental brain tumors. Science 256:1550-1552.
8. Dai Y, Roman M, Naviaux RK, Verma IM: 1992. Gene therapy via primary myoblasts: long-term expression of factor IX protein following transplantation in vivo. Proc Natl Acad Sci USA 89:10,892-10,895.
9. Donahue RE, Kessler SW, Bodine D, et al.: 1992. Helper virus induced T cell lymphoma in nonhuman primates after retroviral mediated gene transfer. J Exp Med 176:1125-1135.
10. Feigner JH, Kumar R, Sridhar CN, et al.: 1994. Enhanced gene delivery and mechanism studies with a novel series of cationic lipid formulations. J Biol Chem 269:2550-2561.
11. Fujiwara T, Grimm EA, Roth JA: 1994. Gene therapeutics and gene therapy of cancer. Curr Opin Oncol 6:96-105.
12. Georges RN, Mukhopadhay T, Zhang Y, Yen N, Roth JA: 1993. Prevention of orthotopic human lung cancer growth by intratracheal instillation of a retroviral antisense K-ras construct. Cancer Res 53:1743-1746.
13. Kaneda Y, Iwai K, Uchida T: 1989. Increased expression of DNA cointroduced with nuclear protein in adult rat liver. Science 243: 375-378.
14. Kasahara N, Dozy AM, Kan YW: 1994. Tissue-specific targeting of retroviral vectors through ligand-receptor interactions. Science 266:1373-1376.
15. Katayose D, Gudas J, Nguyen H, Srivastava S, Cowan KH, Seth P: 1995. Cytotoxic effects of adenovirus-mediated wild-type p53 protein expression in normal and tumor mammary epithelial cells. Clin Cancer Res 1:887-897.
16. Kato K, Kaneda Y, Sakurai M, Nakanishi M, Okada Y: 1991. Direct injection of hepatitis B virus DNA into liver induced hepatitis in adult rats. J Biol Chem 266:22,071-22,074.
17. Kolberg R: 1994. The bystander effect in gene therapy. J NIH Res 6:62-64.
18. Kooyman DL, Byrne GW, McClellan S: 1995. In vivo transfer of GPI-linked complement restriction factors from erythrocytes to endothelium. Science 269:89-92.
19. Ledley FD: 1994. Non-viral gene therapy. Curr Opin Biotechnol 5:626-636.
20. Liu JM, Walsh CE, Nienhuis AW: 1995. Gene therapy of hematologic disorders. In Hoffman R, Benz EJ, Shattil SJ, Furie B, Cohen HJ, Silberstein LE, eds. Hematology, Basic Principles and Practice, 2nd ed. New York, Churchill Livingstone, p 427.
21. Maciejewski JP, Weichold FF, Young NS, et al.: 1995. Intracellular expression of antibody fragments directed against HIV reverse transcriptase prevents HIV infection in vitro. Nat Med 1:667-673.
22. Mulligan RC: 1993. The basic science of gene therapy. Science 260:926-932.
23. Nabel EG: 1995. Gene therapy for cardiovascular disease. Circulation 91:541-548.
24. Nienhuis AW, Walsh CE, Liu JM: 1993. Viruses as therapeutic gene transfer vectors. In Young NS, ed. Viruses and Bone Marrow. New York, Marcel Dekker, p 353.
25. Plautz GE, Yang ZY, Wu BY, Gao X, Huang L, Nabel GJ: 1993. Immunotherapy of malignancy by in vivo gene transfer into tumors. Proc Natl Acad Sci USA 90:4645-4649.
26. Qazilbash MH, Walsh CE, Russell SM, et al.: 1995. Retroviral vector for gene therapy of X-linked severe combined immunodeficiency syndrome. J Hematotherapy 4:91-98.
27. Rosenberg SA, Aebersold P, Cornetta K, et al.: 1990. Gene transfer into humans: immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction. N Engl J Med 323:570-578.
28. Rosenberg SA, Kasid A, Anderson WF, Blaese RM, Abersold P: 1992. Gene therapy of patients with advanced cancer using tumor infiltrating lymphocytes transduced with the gene coding for tumor necrosis factor. Hum Gene Ther 3:57-73.
29. Samulski RJ: 1993. Adeno-associated viruses: integration at a specific chromosomal locus. Curr Opin Genet Dev 3:74-80.
30. Sorrentino BP, Brandt SJ, Bodine D, et al.: 1992. Retroviral transfer of the human MDR1 genes permits selection of drug resistant bone marrow cells in vivo. Science 257:99-103.
31. Trapnell BC, Gorziglia M: 1994. Gene therapy using adenoviral vectors. Curr Opin Biotechnol 5:617-625.
32. Wagner E, Zenke M, Cotten M, Beug H, Birnstiel ML: 1991. Transferrin polycation DNA complexes:the effect of polycations on the structure of the complex and DNA delivery to cells. Proc Natl Acad Sci USA 88:4255-4259.
33. Walsh CE, Grompe M, Vanin E, et al.: 1994a. A functionally active retroviral vector for gene therapy in Fanconi anemia group C. Blood 84:453-459.
34. Walsh CE, Nienhuis AW, Samulski RJ, et al.: 1994b. Phenotypic correction of Fanconi anemia in human hematopoietic cells with a recombinant adeno-associated virus vector. J Clin Invest 94:1440-1448.
35. Weiss RA, Tailor CS: 1995. Retrovirus receptors. Cell 82:531-533.
36. Wu GY, Zhan P, Sze LL, Rosenberg AR, Wu CH: 1994. Incorporation of adenoviruses into a ligand based DNA carrier system results in retention of original receptor specificity and enhances targeted gene expression. J Biol Chem 269:11,548-11,552.
37. Yang Y, Nunes FA, Berenssi K, Furth EE, Gonczol E, Wilson JM: 1994. Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy. Proc Natl Acad Sci USA 91:4407-4411.