New paradigm of gene therapy: Skeletal-muscle-targeting gene therapy for kidney disease

Nephron

Volumn 83, Issue 4, 1999, Pages 296-300

  Imai, Enyu ^a   Isaka, Yoshitaka ^a  

^a OSAKA UNIVERSITY GRADUATE SCHOOL OF MEDICINE   (Japan)

Author keywords

[No Author keywords available]

Indexed keywords

LIPOSOME; SCATTER FACTOR; TRANSFORMING GROWTH FACTOR BETA;

ADENO ASSOCIATED VIRUS; DNA TRANSFECTION; ELECTROPORATION; GENE THERAPY; HUMAN; KIDNEY DISEASE; NONHUMAN; PARAINFLUENZA VIRUS 1; PRIORITY JOURNAL; PROMOTER REGION; SHORT SURVEY; SKELETAL MUSCLE;

EID: 0032736468     PISSN: 00282766     EISSN: None     Source Type: Journal    
DOI: 10.1159/000045420     Document Type: Short Survey

References (29)

1. Japan Society of Dialysis Therapy: An overview of regular dialysis treatment in Japan (as of December 31, 1998). 1999, p 63.
2. Japan Society of Dialysis Therapy: An overview of regular dialysis treatment in Japan (as of December 31, 1998). 1999, p 56.
3. Blease R, Culver K, Miller A, Carter C, Fleisher T, Clerici M, Shearer G, Chang L, Chiang Y, Tolstoshev V: T lymphocyte-derived gene therapy for ADA-SCID: Initial trial after 4 years. Science 1995;270:475-480.
4. Baumgartner I, Pieczek A, Manor O, Blair R, Kearney M, Walsh K, Isner J: Constitutive expression of phVEGF165 after intramuscular gene transfer promotes collateral vessel development in patients with critical limb ischemia. Circulation 1998;97:1114-1123.
5. Bestor T: Methylation meets acetylation. Nature 1998;393:311-312.
6. Wolff J, Malone R, Williams P, Chong W, Acsadi G, Jani A, Felgner P: Direct gene transfer into mouse muscle in vivo. Science 1990; 247:1465-1468.
7. Tripathy S, Svensson E, Black H, Goldwasser E, Margalith M, Hobart P, Leiden J: Long-term expression of erythropoietin in the systemic circulation of mice after intramuscular injection of a plasmic DNA vector. Proc Natl Acad Sci USA 1996;93:10876-10880.
8. Tsurumi Y, Takeshita S, Chen D, Kearney M, Roissow S, Passeri J, Horowitz J, Symes J: Direct intramuscular gene transfer of naked DNA encoding vascular endothelial growth factor augments collateral development and tissue perfusion. Circulation 1996;94:3281-3290.
9. Blezinger P, Wang J, Gondo M, Quezada D, Mehrens D, French M, Singhal A, Sullivan S, Rolland A, Ralston R, Min W: Systemic inhibition of tumor growth and tumor metastasis by intramuscular administration of the endostatin gene. Nat Biotechnol 1999;17:343-348.
10. Tokui M, Takei I, Tashiro F, Shimada A, Kasuga A, Ishii M, Ishii T, Takatsu K, Saruta T, Miyazaki J: Intramuscular injection of expression plasmid DNA is an effective means of long-term systemic delivery of interleukin-5. Biochem Biophys Res Commun 1997;233: 527-531.
11. Niwa H, Yamamoto K, Miyazaki J: Efficient selection for high-expression transfectants with a novel eukaryotic vector. Gene 1991;108:193-199.
12. Akagi Y, Isaka Y, Akagi A, Ikawa M, Takenaka M, Moriyama T, Yamauchi A, Horio M, Ueda N, Okabe M, Imai E: Transcriptional activation of a hybrid promoter composed of cytomegalovirus enhancer and β-actin/β-globin gene in glomerular epithelial cells. Kidney Int 1997;51:1265-1269.
13. Anwer K, Shi M, French M, Muler S, Chen W, Liu Q, Proctor B, Wang J, Mumper R, Singhal A, Rolland A, Alila H: 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.
14. Li X, Eastman E, Schwartz R, Draghia-Akli R: Synthetic muscle promoters: Activities exceeding naturally occurring regulatory sequences. Nat Biotechnol 1999;17:241-245.
15. Saeki Y, Matsumoto N, Nakano Y, Mori M, Awai K, Kaneda Y: Development and characterization of cationic liposomes conjugated with HVJ (Sendai virus): Reciprocal effect of cationic lipid for in vitro and in vivo gene transfer. Hum Gene Ther 1997;8:2133-2141.
16. Aihara H, Miyazaki J: Gene transfer into muscle by electroporation in vivo. Nat Biotechnol 1998;16:867-870.
17. Mir L, Bureau M, Gehl J, Rangara R, Rouy D, Caillaud J, Delaere P, Branellec D, Schwartz B, Scherman D: High efficiency gene transfer into skeletal muscle mediated by electric pulses. Proc Natl Acad Sci USA 1999;96:4262-4267.
18. Kaplitt M, Leone P, Samulski R, Xiao X, Pfaff D, O'Malley K, During M: Long-term gene expression and phenotypic correction using adeno-associated virus vector in the mammalian brain. Nat Genet 1994;8:148-154.
19. Ponnazhagan S, Erickson D, Kearns W, Zhou S, Nahreini P, Wang X, Srivastava A: Lack of site-specific integration of the recombinant adeno-associated virus 2 genomes in human cells. Hum Gene Ther 1997;8:275-284.
20. Kessler P, Podsakoff G, Chen X, McQuiston S, Colosi P, Matelis L, Kurtzman G, Byrne B: Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc Natl Acad Sci USA 1996;93:14082-14087.
21. Ye X, Rivera V, Zoltick P, Cerasoli F Jr, Schnell M, Gao G, Hughes J, Gilman M, Wilson J: Regulated delivery of therapeutic proteins after in vivo somatic cell gene. Science 1999;283:88-91.
22. Isaka Y, Brees DK, Ikegaya K, Kaneda Y, Imai E, Noble NA, Border WA: Gene therapy by skeletal muscle expression of decorin prevents fibric disease in rat kidney. Nat Med 1996;2: 418-423.
23. Isaka Y, Akagi Y, Ando Y, Tsujie M, Sudo T, Ohno N, Border W, Noble N, Kaneda Y, Hori M, Imai E: Gene therapy by transforming growth factor-β receptor-IgG Fc chimera suppressed extracellular matrix accumulation in experimental glomerulonephritis. Kidney Int 1999;55:465-475.
24. Montesano R, Matsumoto K, Nakamura T, Orci L: Identification of a fibroblast-derived epithelial morphogen as hepatocyte growth factor. Cell 1991;67:901-908.
25. Tsarfaty I, Rong J, Rulong S, da Silva P, Vande Woude G: The Met proto-oncogene mesenchymal to epithelial cell conversion. Science 1994; 263:98-101.
26. Liu Y, Sun A, Dworkin L: Hepatocyte growth factor protects renal epithelial cells from apoptotic death. Biochem Biophys Res Commun 1998;246:821-826.
27. Kawaida K, Matsumoto K, Shimizu H, Nakamura T: Hepatocyte growth factor prevents acute renal failure and accelerates renal regeneration in mice. Proc Natl Acad Sci USA 1994; 91:4357-4361.
28. Mizuno S, Kurosawa T, Matsumoto K, Mizuno-Horikawa Y, Okamoto M, Nakamura T: Hepatocyte growth factor prevents renal fibrosis and dysfunction in a mouse model of chronic renal diseases. J Clin Invest 1998;101: 1827-1834.
29. Ueki T, Kaneda Y, Tsutsui H, Nakanishi K, Sawa Y, Morishita R, Matsumoto K, Nakamura T, Takahashi H, Okamoto E, Fujimoto J: Hepatocyte growth factor gene therapy of liver cirrhosis in rats. Nat Med 1999;5:226-230.