Lyophilization of cationic lipid-protamine-DNA (LPD) complexes

Journal of Pharmaceutical Sciences

Volumn 89, Issue 3, 2000, Pages 355-364

  Li, Bei ^a,d   Li, Song ^b   Tan, Yadi ^b   Stolz, Donna B ^c   Watkins, Simon C ^c   Block, Lawrence H ^a   Huang, Leaf ^b  

^a DUQUESNE UNIVERSITY   (United States)

^b UNIVERSITY OF PITTSBURGH   (United States)

^c UNIVERSITY OF PITTSBURGH SCHOOL OF MEDICINE   (United States)

^d UNIVERSITY OF KANSAS   (United States)

Author keywords

Gene therapy; Lipopolyplex; Lyophilization; Nonviral vector

Indexed keywords

CHOLESTEROL; DNA; LIPOSOME; PROTAMINE SULFATE;

ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; DNA DRUG COMPLEX; ESCHERICHIA COLI; FEMALE; FREEZE DRYING; FREEZING; GENE THERAPY; NONHUMAN; PARTICLE SIZE; PLASMID; TEMPERATURE SENSITIVITY; THAWING;

EID: 0034098765     PISSN: 00223549     EISSN: None     Source Type: Journal    
DOI: 10.1002/(SICI)1520-6017(200003)89:3<355::AID-JPS7>3.0.CO;2-H     Document Type: Article

References (34)

1. Miller AD. 1992. Human gene therapy comes of age. Nature 357:455-460.
2. Ross G, Erickson R, Knorr D, Motulsky AG, Parkman R, Samulski J, Straus SE, Smith BR. 1996. Gene therapy in the United States: a 5 year status report. Hum Gene Ther 7:1781-1790.
3. Herz J, Gerard RD. 1993. Adenovirus mediated transfer of low density lipoprotein receptor gene acutely accelerates cholesterol clearance in normal mice. Proc Natl Acad Sci USA 90:2812-2816.
4. Simon RH, Engelhardt JF, Yang Y, Zepeda M, Weber-Pendleton S, Grossman M, Wilson JM. 1993. Adenovirus mediated transfer of the CFTR gene to lung of nonhuman primates: toxicity study. Hum Gene Ther 4:771-780.
5. Ali M, Lemoine NR, Ring CJA. 1994. The use of DNA virus as vectors for gene therapy. Gene Ther 1:367-384.
6. Gao X, Huang L. 1995. Cationic liposome mediated gene transfer. Gene Ther 2:710-722.
7. Felgner PL. 1996. Improvements in cationic liposomes for in vivo gene transfer. Hum Gene Ther 7:1791-1793.
8. Wu GY, Wu CH. 1988. Receptor mediated gene delivery and expression in vivo. J Biol Chem 263: 14621-14624.
9. Boussif O, Lezoualc'h F, Zanta MA, Mergny MD, Scherman D, Demeneix B, Behr JP. 1995. A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. Proc Natl Acad Sci USA 92:7297-7301.
10. Felgner PL, Gadek TR, Holm M, Roman R, Chan HW, Wenz M, Northrop JP, Ringold GM, Danielsen M. 1987. Lipofection: a highly efficient, lipid mediated DNA transfection procedure. Proc Natl Acad Sci USA 84:7413-7417.
11. Nabel GJ, Nabel EG, Yang ZY, Fox BA, Plautz GE, Gao X, Huang L, Shu S, Gordon D, Chang AE. 1993. Direct gene transfer with DNA liposome complexes in melanoma: expression, biological activity, and lack of toxicity in humans. Proc Natl Acad Sci USA 90:11307-11311.
12. Caplen NJ, Alton EWFW, Middleton PG, Dorin JR, Stevenson BJ, Gao X, Durham SR, Jeffrey PK, Hodson ME, Coutelle C, Huang L, Porteous DJ, Williamson R, Geddes DM. 1995. Liposome mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Nat Med 1:39-46.
13. McLachlan G, Ho L-P, Davidson-Smith H, Samways J, Davidson H, Stevenson BJ, Carothers AD, Alton EWFW, Middleton PG, Smith SN, Kallmeyer G, Michaelis U, Seeber S, Naujoks K, Greening AP, Innes JA, Dorin JR, Porteous DJ. 1996. Laboratory and clinical studies in support of cystic fibrosis gene therapy using pCMVCFTRDOTAP. Gene Ther 3:1113-1123.
14. Middaugh CR, Evans RK, Montgomery DL, Casimiro DR. 1998. Analysis of plasmid DNA from a pharmaceutical perspective. J Pharm Sci 87: 130-146.
15. Felgner PL, Tsai YJ, Sukhu L, Wheeler CJ, Manthorpe M, Marshall J, Cheng SH. 1995. Improved cationic lipid formulations for in vivo gene therapy. Ann NY Acad Sci 1995;772:126-139.
16. Gustafsson J, Arvidson G, Karlsson G, Almgren M. 1995. Complexes between cationic liposomes and DNA visualized by Cryo-TEM. Biochim Biophys Acta 1235:305-312.
17. Nabel GJ, Chang A, Nabel EG, Plautz G, Fox BA, Huang L, Shu S. 1997. Immunotherapy of malignancy by in vivo gene transfer into tumors. Hum Gene Ther 3:399-410.
18. Anchordoquy TJ, Carpenter JF, Kroll DJ. 1997. Maintenance of transfection rates and physical characterization of Lipid/DNA complexes after freeze-drying and rehydration. Arch Biochem Biophys 348:199-206.
19. Cherng JY, Van de Wetering P, Talsma H, Crommelin DJA, Hennink WE. 1997. Freeze-drying of poly((2-dimethylamino) ethyl methacrylate)-based gene delivery systems. Pharm Res 14:1838-1841.
20. Talsma H, Cherng JY, Lehrmann H, Kursa M, Ogris M, Hennink WE, Cotton M, Wagner E. 1997. Stabilization of gene delivery systems by freeze-drying. Int J Pharm 157:233-238.
21. Gao X, Huang L. 1996. Potentiation of cationic liposome-mediated gene delivery by polycations. Biochemistry 35:1027-1036.
22. Li S, Huang L. 1997. In vivo gene transfer via intravenous administration of cationic lipid-protamine-DNA (LPD) complexes. Gene Ther 4: 891-900.
23. Li S, Rizzo MA, Bhattacharya S, Huang L. 1998. Characterization of cationic lipid-protamine-DNA (LPD) complexes for intravenous gene delivery. Gene Ther 5:930-937.
24. Prensky W. 1976. The radioiodination of RNA and DNA to high specific activities. Cell Biol 13: 121-152.
25. Li S, Huang L. 1999. Novel lipidic vectors for gene transfer. In: Huang L, Hung M-C, Wagner E, editors. Nonviral vectors for gene therapy. San Diego: Academic Press, p 290-309.
26. Crowe JH, Crowe LM. 1992. Preservation of liposomes by freeze-drying. In: Gregoriadis G, editor. Liposome technology, Vol I, Liposome preparation and related techniques. 2nd edition. Boca Raton: CRC Press, p 229-252.
27. Madden TD, Boman N. 1999. Lyophilization of liposomes. In: Janoff AS, editor. Liposomes: Rational design. New York: Marcel Dekker, Inc. pp 261-282.
28. Anchordoguy TJ, Rudolph AS, Carpenter JF, Crowe JH. 1987. Modes of interaction of cryoprotectants with membrane phospholipids during freezing. Cryobiology 24:324-331.
29. Sternberg B. 1998. Ultrastructural morphology of cationic liposome-DNA complexes for gene therapy. In: Lasic DD, Papahadjopoulos D, editors. Medical applications of liposomes. New York: Elsevier. pp 395-427.
30. Ross PC, Su H. 1999. Lipoplex size is a major determinant of in vitro lipofection efficiency. Gene Ther 6:643-650.
31. Liu Y, Mounkes LC, Liggitt HD, Brown CS, Soludin I, Heath TD, Debs RJ. 1997. Factors influencing the efficiency of cationic liposome-mediated intravenous gene delivery. Nat Biotechnol 15: 167-173.
32. Harrigan PR, Madden TD, Cullis PR. 1990. Protection of liposomes during dehydration or freezing. Chem Phys Lipids 52:139-149.
33. Anchordoquy TJ, Girouard LG, Carpenter JF, Kroll DJ. 1998. Stability of lipid/DNA complexes during agitation and freeze-thawing. J Pharm Sci 87:1046-1051.
34. Liu F, Yang JP, Huang L, Liu D. 1996. New cationic lipid formulations for gene transfer. Pharm Res 13:1856-1860.