Nonviral gene therapy: Promises and challenges

Gene Therapy

Volumn 7, Issue 1, 2000, Pages 31-34

  Li, S ^a   Huang, L ^a  

^a UNIVERSITY OF PITTSBURGH   (United States)

Author keywords

Gene therapy; Gene transfer; Nonviral vector

Indexed keywords

LIPID; PLASMID DNA;

CHIMERA; CLINICAL PRACTICE; CLINICAL TRIAL; CPG ISLAND; EXPRESSION VECTOR; GENE THERAPY; GENE TRANSFER; GENETIC TRANSFECTION; HUMAN; NONHUMAN; PRIORITY JOURNAL; SHORT SURVEY;

EID: 0033972869     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3301110     Document Type: Short Survey

References (26)

1. Wolff JA et al. Direct gene transfer into mouse muscle in vivo. Science 1990; 247: 1465-1468.
2. Ulmer JB et al. Heterologous protection against influenza by injection of DNA encoding a viral protein. Science 1993; 259: 1745-1749.
3. Condon C et al. DNA-based immunization by in vivo transfection of dendritic cells. Nature Med 1996; 2: 1122-1128.
4. Liu F, Song Y, Liu D. Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA. Gene Therapy 1999; 6: 1258-1266.
5. Yang NS et al. In vivo and in vitro gene transfer to mammalian somatic cells by particle bombardment. Proc Natl Acad Sci USA 1990; 87: 9568-9572.
6. Rols MP et al. In vivo electrically mediated protein and gene transfer in murine melanoma. Nat Biotechnol 1998; 16: 168-171.
7. Rizzuto G et al. Efficient and regulated erythropoietin production by naked DNA injection and muscle electroporation. Proc Natl Acad Sci USA 1999; 96: 6417-6422.
8. Anwer K et al. Systemic effect of human growth hormone after intramuscular injection of a single dose of a muscle-specific gene medicine. Hum Gene Ther 1998; 9: 659-670.
9. Feigner PL et al. Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure. Proc Natl Acad Sci USA 1987; 84: 7413-7417.
10. Wheeler CJ et al. A novel cationic lipid greatly enhances plasmid DNA delivery and expression in mouse lung. Proc Natl Acad Sci USA 1996; 93: 11454-11459.
11. Lee ER et al. Detailed analysis of structures and formulations of cationic lipids for efficient gene transfer to the lung. Hum Gene Ther 1996; 7: 1701-1717.
12. Nabel GJ et al. Direct gene transfer with DNA-liposome complexes in melanoma: expression, biologic activity, and lack of toxicity in humans. Prof Natl Acad Sci USA 1993; 90: 11307-11311.
13. Caplen NJ et al. Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Nat Med 1995; 1: 39-46.
14. Radler JO, Koltover I, Salditt T, Safinya CR. Structure of DNA-cationic liposome complexes: DNA intercalation in multilamellar membranes in distinct interhelical packing regimes. Science 1997; 275: 810-814.
15. Subramanian A, Ranganathan P, Diamond SL. Nuclear targeting peptide scaffolds for lipofection of nondividing mammalian cells. Nat Biotechnol 1999; 17: 873-877.
16. Li S et al. Dynamic changes in the characteristics of cationic lipidic vectors after exposure to mouse serum: implications for intravenous lipofection. Gene Therapy 1999; 6: 585-594.
17. Gao X, Huang L. Potentiation of cationic liposome-mediated gene delivery by polycations. Biochemistry 1996; 35: 1027-1036.
18. Liu Y et al. Factors influencing the efficiency of cationic liposome-mediated intravenous gene delivery. Nat Biotechnol 1997; 15: 167-173.
19. Boussif O et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. Proc Natl Acad Sci USA 1995; 92: 7297-7301.
20. Roy K, Mao HQ, Huang SK, Leong KW. Oral gene delivery with chitosan-DNA nanoparticles generates immunologic protection in a murine model of peanut allergy. Nat Med 1999; 5: 387-391.
21. Cole-Strauss et al. Correction of the mutation responsible for sickle cell anemia by an RNA-DNA oligonucleotide. Science 1996; 273: 1386-1389.
22. Kren BT et al. Correction of the UDP-glucuronosyltransferase gene defect in the Gunn rat model of Crigler-Najjar syndrome type I with a chimeric oligonucleotide. Proc Natl Acad Sci USA 1999; 96: 10349-10354.
23. Yew NS et al. Contribution of plasmid DNA to inflammation in the lung after administration of cationic lipid:pDNA complexes. Hum Gene Ther 1999; 10: 223-234.
24. Li S et al. Effect of immune response on gene transfer to the lung via systemic administration of cationic lipidic vectors. Am J Physiol 1999; 276: L796-L804.
25. Krieg AM et al. CpG motifs in bacterial DNA trigger direct B-cell activation. Nature 1995; 374: 546-549.
26. Chu RS et al. CpG oligodeoxy-nucleoticles act as adjuvants that switch on T helper 1 (Th1) immunity. J Exp Med 1997; 186: 1623-1631.