High-efficiency plasmid gene transfer into dystrophic muscle

Gene Therapy

Volumn 10, Issue 6, 2003, Pages 504-512

  Gollins, H ^a   McMahon, J ^a   Wells, K E ^a   Wells, D J ^a  

^a CHARING CROSS HOSPITAL   (United Kingdom)

Author keywords

Electroporation; Fibres; Gene therapy; Hyaluronidase; Muscular dystrophy; Plasmid; Skeletal muscle

Indexed keywords

BETA GALACTOSIDASE; DYSTROPHIN; HYALURONIDASE; PLASMID DNA;

AGE; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; CONNECTIVE TISSUE; CONTROLLED STUDY; DRUG EFFICACY; DUCHENNE MUSCULAR DYSTROPHY; ELECTRIC FIELD; FEMALE; GENE DELIVERY SYSTEM; GENE TRANSFER; GENETIC TRANSFECTION; MALE; MOUSE; MUSCLE INJURY; NONHUMAN; PHENOTYPE; PLASMID; PRIORITY JOURNAL; REVIEW; SEX DIFFERENCE;

ANIMALS; BETA-GALACTOSIDASE; DYSTROPHIN; ELECTROPORATION; FEMALE; GENE EXPRESSION; GENE THERAPY; INJECTIONS, INTRAMUSCULAR; MALE; MICE; MICE, INBRED C57BL; MICE, INBRED CBA; MICE, INBRED MDX; MUSCLE, SKELETAL; MUSCULAR DYSTROPHIES; PLASMIDS;

ANIMALIA;

EID: 0037348637     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3301927     Document Type: Review

References (28)

1. Acsadi G et al. Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs. Nature 1991; 352: 815-881.
2. Wolff JA et al. Direct gene transfer into mouse muscle in vivo. Science 1990; 247(Part 1): 1465-1468.
3. Davis HL, Whalen RG, Demeneix BA. Direct gene transfer into skeletal muscle in vivo: factors affecting efficiency of transfer and stability of expression. Hum Gene Ther 1993; 4: 151-159.
4. Levy MY et al. Characterization of plasmid DNA transfer into mouse skeletal muscle: evaluation of uptake mechanism expression and secretion of gene products into blood. Gene Ther 1996; 3: 201-211.
5. Manthorpe M et al. Gene therapy by intramuscular injection of plasmid DNA: studies on firefly luciferase gene expression in mice. Hum Gene Ther 1993; 4: 419-431.
6. Wells DJ, Goldspink G. Age and sex influence expression of plasmid DNA directly injected into mouse skeletal muscle. FEBS Lett 1992; 306: 203-205.
7. Wells DJ et al. Evaluation of plasmid DNA for in vivo gene therapy: factors affecting the number of transfected fibers. J Pharm Sci 1998; 87: 763-768.
8. Mathiesen I. Electropermeabilization of skeletal muscle enhances gene transfer in vivo. Gene Ther 1999; 6: 508-514.
9. Mir LM et al. Long-term, high level in vivo gene expression after electric pulse-mediated gene transfer into skeletal muscle. C R Acad Sci III 1998; 321: 893-899.
10. Aihara H, Miyazaki J. Gene transfer into muscle by electroporation in vivo. Nat Biotechnol 1998; 16: 867-870.
11. Vilquin J et al. Electrotransfer of naked DNA in the skeletal muscles of animal models of muscular dystrophies. Gene Ther 2001; 8: 1097-1107.
12. McMahon JM et al. Optimisation of electrotransfer of plasmid into skeletal muscle by pretreatment with hyaluronidase - increased expression with reduced muscle damage. Gene Ther 2001; 8: 1264-1270.
13. Vicat JM et al. Muscle transfection by electroporation with high-voltage and short-pulse currents provides high-level and long-lasting gene expression. Hum Gene Ther 2000; 11: 909-916.
14. Mennuni C et al. Hyaluronidase increases electrogene transfer efficiency in skeletal muscle. Hum Gene Ther 2002; 13: 355-365.
15. Danko I et al. High expression of naked plasmid DNA in muscles of young rodents. Hum Mol Genet 1997; 6: 1435-1443.
16. Jooss K, Turka LA, Wilson JM. Blunting of immune responses to adenoviral vectors in mouse liver and lung with CTLA4Ig. Gene Ther 1998; 5: 309-319.
17. Halbert CL et al. Successful readministration of adeno-associated virus vectors to the mouse lung requires transient immunosuppression during the initial exposure. J Virol 1998; 72: 9795-9805.
18. Mir LM et al. High-efficiency gene transfer into skeletal muscle mediated by electric pulses. Proc Natl Acad Sci USA 1999; 96: 4262-4267.
19. Coulton GR et al. The mdx mouse skeletal muscle myopathy: I. A histological, morphometric and biochemical investigation. Neuropathol Appl Neurobiol 1988; 14: 53-70.
20. Budker V et al. Hypothesis: naked plasmid DNA is taken up by cells in vivo by a receptor-mediated process. J Gene Med 2000; 2: 76-88.
21. Satkauskas S et al. Slow accumulation of plasmid in muscle cells: supporting evidence for a mechanism of DNA uptake by receptor-mediated endocytosis. Mol Ther 2001; 4: 317-323.
22. Somiari S et al. Theory and in vivo application of electroporative gene delivery. Mol Ther 2000; 2: 178-187.
23. Vincent N et al. Long-term correction of mouse dystrophic degeneration by adenovirus-mediated transfer of a minidystrophin gene. Nat Genet 1993; 5: 130-134.
24. Deconinck N et al. Functional protection of dystrophic mouse (mdx) muscles after adenovirus-mediated transfer of a dystrophin minigene. Proc Natl Acad Sci USA 1996; 93: 3570-3574.
25. Clemens PR et al. In vivo muscle gene transfer of full-length dystrophin with an adenoviral vector that lacks all viral genes. Gene Ther 1996; 3: 965-972.
26. Jiao S et al. Direct gene transfer into nonhuman primate myofibers in vivo. Hum Gene Ther 1992; 3: 21-33.
27. Ferrer A. PhD thesis, University of London, 2001.
28. Ferrer A, Wells KE, Wells DJ. Immune responses to dystropin: implications for gene therapy of Duchenne muscular dystrophy. Gene Ther 2000; 7: 1439-1446.