Targeted Delivery of Plasmid DNA to Myogenic Cells via Transferrin-Conjugated Peptide Nucleic Acid

Molecular Therapy

Volumn 1, Issue 3, 2000, Pages 236-243

  Liang, Kenneth W ^a   Hoffman, Eric P ^b   Huang, Leaf ^a  

^a UNIVERSITY OF PITTSBURGH   (United States)

^b CHILDREN'S NATIONAL MEDICAL CENTER   (United States)

Author keywords

Gene therapy; nonviral vector; targeted delivery; myogenic cells; PNA

Indexed keywords

DISULFIDE; LIGAND; LUCIFERASE; PEPTIDE NUCLEIC ACID; TRANSFERRIN; TRANSFERRIN RECEPTOR;

ARTICLE; CELL LINE; CHEMISTRY; CONFORMATION; CYTOLOGY; ENDOCYTOSIS; GENETIC TRANSFECTION; GENETICS; METABOLISM; METHODOLOGY; PHYSIOLOGY; PLASMID; REPORTER GENE; SKELETAL MUSCLE;

CELL LINE; DISULFIDES; ENDOCYTOSIS; GENES, REPORTER; LIGANDS; LUCIFERASES; MUSCLE, SKELETAL; NUCLEIC ACID CONFORMATION; PEPTIDE NUCLEIC ACIDS; PLASMIDS; RECEPTORS, TRANSFERRIN; TRANSFECTION; TRANSFERRIN;

EID: 0034154411     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1006/mthe.2000.0043     Document Type: Article

References (49)

1. Acsadi G., Jani A., Massie B., Simoneau M., Holland P., Blaschuk K., Karpati G. A differential efficiency of adenovirus-mediated in vivo gene transfer into skeletal muscle cells of different maturity. Hum. Mol. Genet. 3:1994;579-584.
2. Thomason D. B., Booth F. W. Stable incorporation of a bacterial gene into adult rat skeletal muscle in vivo. Am. J. Physiol. 258:1990;C578-C581.
3. Li J., Dressman D., Tsao Y. P., Sakamoto A., Hoffman E. P., Xiao X. RAAV vector-mediated sarcoglycan gene transfer in a hamster model for limb girdle muscular dystrophy. Gene Ther. 6:1999;74-82.
4. Wolff J. A., Malone R. W., Williams P., Chong W., Acsadi G., Jani A., Felgner P. L. Direct gene transfer into mouse muscle in vivo. Science. 247:1990;1465-1468.
5. Wolff J. A., Dowty M. E., Jiao S., Repetto G., Berg R. K., Ludtke J. J., Williams P., Slautterback D. B. Expression of naked plasmids by cultured myotubes and entry of plasmids into T tubules and caveolae of mammalian skeletal muscle. J. Cell Sci. 103:1992;1249-1259.
6. Levy M. Y., Barron L. G., Meyer K. B., Szoka F. C. Jr. Characterization of plasmid DNA transfer into mouse skeletal muscle: Evaluation of uptake mechanism, expression and secretion of gene products into blood. Gene Ther. 3:1996;201-211.
7. Hartikka J., Sawdey M., Cornefert-Jensen F., Margalith M., Barnhart K., Nolasco M., Vahlsing H. L., Meek J., Marquet M., Hobart P., Norman J., Manthorpe M. An improved plasmid DNA expression vector for direct injection into skeletal muscle. Hum. Gene Ther. 7:1996;1205-1217.
8. Wells D. J. Intramuscular injection of plasmid DNA. Mol. Cell Biol. Hum. Dis. Ser. 5:1995;83-103.
9. Mumper R. J., Wang J., Klakamp S. L., Nitta H., Anwer K., Tagliaferri F., Rolland A. P. Protective interactive noncondensing (PINC) polymers for enhanced plasmid distribution and expression in rat skeletal muscle. J. Controlled Release. 52:1998;191-203.
10. Hoffman E. P., Kunkel L. M., Angelini C., Clarke A., Johnson M., Harris J. B. Improved diagnosis of Becker muscular dystrophy by dystrophin testing. Neurology. 39:1989;1011-1017.
11. Hoffman E. P., Arahata K., Minetti C., Bonilla E., Rowland L. P. Dystrophinopathy in isolated cases of myopathy in females. Neurology. 42:1992;967-975.
12. Pegoraro E., Schimke R. N., Garcia C., Stern H., Hoffman E. P. Genetic and biochemical normalization in female carriers of Duchenne muscular dystrophy: Evidence for failure of dystrophin production in dystrophin-competent myonuclei. Neurology. 45:1995;677-690.
13. Acsadi G., Dickson G., Love D. R., Jani A., Walsh F. S., Gurusinghe A., Wolff J. A., Davies K. E. Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs. Nature. 352:1991;815-818.
14. Perales J. C., Ferkol T., Molas M., Hanson R. W. An evaluation of receptor-mediated gene transfer using synthetic DNA-ligand complexes. Eur. J. Biochem. 226:1994;255-266.
15. Wang C. Y., Huang L. pH-sensitive immunoliposomes mediate target-cell-specific delivery and controlled expression of a foreign gene in mouse. Proc. Natl. Acad. Sci. USA. 84:1987;7851-7855.
16. Wu G. Y., Wu C. H. Receptor-mediated in vitro gene transformation by a soluble DNA carrier system. J. Biol. Chem. 262:1987;4429-4432.
17. Sebestyen M. G., Ludtke J. J., Bassik M. C., Zhang G., Budker V., Lukhtanov E., Hagstrom J. E., Wolff J. A. DNA vector chemistry: The covalent attachment of signal peptides to plasmid DNA. Natl. Biotech. 16:1998;80-85.
18. Cherny D. Y., Belotserkovskii B. P., Frank-Kamenetskii M. D., Egholm M., Buchardt O., Berg R. H., Nielsen P. E. DNA unwinding upon strand-displacement binding of a thymine-substituted polyamide to double-stranded DNA. Proc. Natl. Acad. Sci. USA. 90:1993;1667-1670.
19. Peffer N. J., Hanvey J. C., Bisi J. E., Thomson S. A., Hassman C. F., Noble S. A., Babiss L. E. Strand-invasion of duplex DNA by peptide nucleic acid oligomers. Proc. Natl. Acad. Sci. USA. 90:1993;10648-10652.
20. Nielsen P. E., Egholm M., Buchardt O. Evidence for (PNA)2/DNA triplex structure upon binding of PNA to dsDNA by strand displacement. J. Mol. Recognit. 7:1994;165-170.
21. Demidov V. V., Yavnilovich M. V., Belotserkovskii B. P., Frank-Kamenetskii M. D., Nielsen P. E. Kinetics and mechanism of polyamide ("peptide") nucleic acid binding to duplex DNA. Proc. Natl. Acad. Sci. USA. 92:1995;2637-2641.
22. Egholm M., Christensen L., Dueholm K. L., Buchardt O., Coull J., Nielsen P. E. Efficient pH-independent sequence-specific DNA binding by pseudoisocytosine-containing bis-PNA. Nucleic Acids Res. 23:1995;217-222.
23. Feero W. G., Li S., Rosenblatt J. D., Sirianni N., Morgan J. E., Partridge T. A., Huang L., Hoffman E. P. Selection and use of ligands for receptor-mediated gene delivery to myogenic cells. Gene Ther. 4:1997;664-674.
24. Wagner E., Zenke M., Cotten M., Beug H., Birnstiel M. L. Transferrin-polycation conjugates as carriers for DNA uptake into cells. Proc. Natl. Acad. Sci. USA. 87:1990;3410-3414.
25. Cotten M., Laengle-Rouault F., Kirlappos H., Wagner E., Mechtler K., Zenke M., Beug H., Birnstiel M. L. Transferrin-polycation-mediated introduction of DNA into human leukemic cells: Stimulation by agents that affect the survival of transfected DNA or modulate transferrin receptor levels. Proc. Natl. Acad. Sci. USA. 87:1990;4033-4037.
26. Boussif O., Lezoualc'h F., Zanta M. A., Mergny M. D., Scherman D., Demeneix B., Behr J. P. A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: Polyethylenimine. Proc. Natl. Acad. Sci. USA. 92:1995;7297-7301.
27. Browning J., Hogg N., Gobe G., Cross R. Capillary density in skeletal muscle of Wistar rats as a function of muscle weight and body weight. Microvasc. Res. 52:1996;281-287.
28. Lee J., Schmid-Schonbein G. W. Biomechanics of skeletal muscle capillaries: Hemodynamic resistance, endothelial distensibility, and pseudopod formation. Ann. Biomed. Eng. 23:1995;226-246.
29. Taylor, A. E., and Granger, D. N.1984. The cardiovascular system; Microcirculation. In Handbook of Physiology (E. M. Renkin, C. C. Michel, and S. R. Geiger, Eds.), pp. 467-520. Am. Physiol. Soc.Bethesda, MD.
30. Karpati G., Acsadi G. The potential for gene therapy in Duchenne muscular dystrophy and other genetic muscle diseases. Muscle Nerve. 16:1993;1141-1153.
31. Budker V., Zhang G., Danko I., Williams P., Wolff J. The efficient expression of intravascularly delivered DNA in rat muscle. Gene Ther. 5:1998;272-276.
32. Zhang G., Vargo D., Budker V., Armstrong N., Knechtle S., Wolff J. A. Expression of naked plasmid DNA injected into the afferent and efferent vessels of rodent and dog livers. Hum. Gene Ther. 8:1997;1763-1772.
33. Budker V., Zhang G., Knechtle S., Wolff J. A. Naked DNA delivered intraportally expresses efficiently in hepatocytes. Gene Ther. 3:1996;593-598.
34. Hickman M. A. Gene expression following direct injection of DNA into liver. Hum. Gene Ther. 5:1994;1477-1483.
35. Song Y. K., Liu F., Liu D. Enhanced gene expression in mouse lung by prolonging the retention time of intravenously injected plasmid DNA. Gene Ther. 5:1998;1531-1537.
36. Meyer K. B. Intratracheal gene delivery to the mouse airway: Characterization of plasmid DNA expression and pharmacokinetics. Gene Ther. 2:1995;450-460.
37. Tsan M. F., White J. E., Shepard B. Lung-specific direct in vivo gene transfer with recombinant plasmid DNA. Am. J. Physiol. 268:1995;11052-11056.
38. Lin H. Expression of recombinant genes in myocardium in vivo after direct injection of DNA. Circulation. 82:1990;2217-2221.
39. Sikes M. L., O'Malley B. W., Finegold M. J., Ledley F. D. In vivo gene transfer into rabbit thyroid follicular cells by direct DNA injection. Hum. Gene Ther. 5:1994;837-844.
40. Yang J. P., Huang L. Direct gene transfer to mouse melanoma by intratumoral injection of free DNA. Gene Ther. 3:1996;542-548.
41. Vile R. G., Hart I. R. Use of tissue-specific expression of the herpes simplex virus thymidine kinase gene to inhibit growth of established murine melanomas following direct intratumoral injection of DNA. Cancer Res. 53:1993;3860-3864.
42. Egholm M., Buchardt O., Nielsen P., Berg R. H. Peptide nucleic acids (PNA): Oligonucleotide analogues with an achival peptide backbone. J. Am. Chem. Soc. 114:1992;1895-1897.
43. Egholm M., Nielsen P., Buchardt O., Berg R. H. Recognition of guanine and adenine in DNA by cytosine and thymidine containing peptide nucleic acids (PNA). J. Am. Chem. Soc. 114:1992;9677-9678.
44. Nielsen, P. E., Egholm, M., Berg, R. H., and Buchardt, O.1993. Antisense Research and Applications (S. T. Crooke, and B. Lebleu, Eds.), pp. 363-374. CRC Press, Boca Raton, FL.
45. Rippe B., Haraldsson B. Transport of macromolecules across microvascular walls: The two-pore theory. Physiol. Rev. 74:1994;163-219.
46. Bentin T., Nielsen P. E. Enhanced peptide nucleic acid binding to supercoiled DNA: Possible implications for DNA "breathing" dynamics. Biochemistry. 35:1996;8863-8869.
47. Zelphati O., Liang X., Hobart P., Felgner P. L. Gene chemistry: Functionally and conformationally intact fluorescent plasmid DNA. Hum. Gene Ther. 10:1999;15-24.
48. Cheng P. W. Receptor ligand-facilitated gene transfer: enhancement of liposome-mediated gene transfer and expression by transferrin. Hum. Gene Ther. 7:1996;275-282.
49. Kurrle A., Rieber P., Sackman E. Reconstitution of transferrin receptor in mixed lipid vesicles: An example of the role of elastic and electrostatic forces for protein/lipid assembly. Biochemistry. 29:1990;8274-8282.