2002, Number 4
<< Back Next >>
Ann Hepatol 2002; 1 (4)
Genomic medicine in Mexico. Applications of gene therapy for cirrhosis reversion
Armendariz-Borunda J
Language: English
References: 28
Page: 169-174
PDF size: 54.50 Kb.
Text Extraction
Genomic medicine represents a powerful armamentarium to tackle down most of chronic diseases which have not, so far, defeated. Thus, this new and powerful biotechnologic set of weapons enable us to make use of molecular diagnostic to detect silent diseases, otherwise undetectable by conventional analysis. Moreover, elucidation of the complete and final draft of the human genome code will allow, although not in this decade, the design of specific farmaco-genetic treatments for patients on basis of their individual genetic code. Regarding new medical treatments, gene therapy as emerged as a true hope for treatment of many chronic diseases. 636 FDA-.approved clinical protocols are currently undergoing and sooner than later we´ll be witness of the results.
REFERENCES
Gene therapy clinical trials. The journal of gene medicine. www.wiley.co.uk/genmed.
Kay M, Liu D, Hoogerbrugge P. Gene Therapy. Proc Natl Acad Sci USA 1997; 94: 12744-12746.
Hacein-Bey- S, Le Deist F, Carlier F, Bouneaud C, Christophe Hue Ch, De Villartay J, Thrasher A, et. al. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N Eng J Med April 2002; 346(16): 1185-1193.
Kay M, Manno C, Ragni M, Larson P, Couto L, Mcclelland A, Glader B, et al. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nature Genetics 2000; 24: 257-261.
Hacein-Bey FF, Cavazzana-Calvo M. Gene theraphy of severe combined immunodeficiencies. Nat Rev Immunol 2002; 2(8): 615-21.
Porada Ch, Tran N, Eglitis M, Moen R, Troutman L, Flake A, Zhao Y, et al. In utero gene therpay: Transfer and long-term expression of the bacterial neo gene in sheep after direct injection of retroviral vectors into preimmune fetuses. Human Gene Therapy 1998; 9: 1571-1585.
Zanjani E, Anderson W. Prospects for in utero human gene therapy. Science 1999; 285: 2084-2088.
Zern M, Kresina T. Hepatic drug delivery and gene therapy. Hepatology 1997; 25(2): 484-491.
Ferry N, Heard J. Liver-directed gene transfer vectors. Human Gene Therapy 1998; 9: 1975-1978.
Martinez-Fong D, Mullersman J, Purchio A, Armendariz-Borunda J, Martinez-Hernandez A. Nonenzymatic glycosylation of poly-L-lisine: A new tool for targeted gene delivery. Hepatology 1994; 20(6): 1602-1608.
De Godoy J, Malafosse R, Fabre M, Mitchell C, Mehtali M, Houssin D, Soubrane O. A preclinical model of hepatocyte gene transfer: the in vivo, in situ perfused rat liver. Gene Therapy 2000; 7: 1816-1823.
Bataller R, Brenner D. Hepatic stellate cells as a target for the treatment of liver fibrosis. Seminars in Liver Disease 2001; 21(3): 437-451.
Kafr T. Lentivirus vectors: difficulties and hopes before clinical trials. Curr Opin Mol Ther Aug. 2001; 3(4): 316-26.
Russell D, Miller AD. Foamy virus vectors. Journal of Virology 1996; 70(1): 217-222.
Olivarez E, Hollis R, Chalberg T, Meuse L, Kay M, Calos M. Site specific genomic integration produces therapeutic factor IX levels in mice. Nature Biotechnology 2001; 20: 1124-1128.
Stephen S, Ehrhardt, A, Giehm J, Meuse L, Pham T, Kay M. Transposition from a gutless adeno-transposon vector stabilizes transgene expression in vivo. Nature Biotechnology 2002; 20: 999-1005.
Hartigan-O´Connor D, Amalafitano A, Chamberlain J. Improved production of gutted adenovirus in cells expressing adenovirus preterminal protein and DNA polymerase. Journal of Virology 1999; 73(9): 7835-7841.
Liu F, Huang L. Development of non-viral vectors for systemic gene delivery. Journal of Controlled Release 2002; 78: 259-266.
Rudolph KL, Chang S, Millard M, Schreiber-Agus N, DePinho RA. Inhibition of experimental liver cirrhosis in mice by telomerase gene delivery. Science 2000; 287: 1253-1258.
Yu, Q., Shao, R., Quian, HS, George SE, Rockey DC. Gene transfer of the neuronal NO synthase isoform to cirrhotic rat liver ameliorates portal hypertension. J Clin Invest 2000; 105(6): 741-748.
UesugiT, Froh M, Arteel GE, Bradford BU, Gabele E, Wheeler MD, Thurman RG. Delivery of IkB superrepresor gene with adenovirus reduces early alcohol- induced liver injury in rats. Hepatology 2001; 34: 1149-1157.
Hecht N, Pappo O, Shouval D, Rose-John S, Galun E, Axelrod JH. Hyper-IL-6 gene therapy reverses fulminant hepatic failure. Molecular Therapy 2001; 3(5): 683-687.
Qi Z, Atsuchi N, Ooshima A, Takeshita A, Ueno H. Blockade of type beta transforming growth factor signaling prevents liver fibrosis and dysfunction in the rat. Proc Natl Acad Sci USA 1999; 96: 2345-2349.
Aguilar L, Aguilar-Cordova E, Ando D, Armendariz-Borunda J, Brud P, Butler E, Carter B, et al. A Prescription for gene therapy. Molecular Therapy 2000; 1(5): 385-388.
Garcia-Bañuelos J, Siller-Lopez F, Miranda A, Aguilar LK, Aguilar-Cordova E, Armendariz-Borunda J. Cirrhotic rat livers with extensive fibrosis can be safely transduced with clinical-grade adenoviral vectors. Evidence of cirrhosis reversion. Gene Therapy 2002; 9: 127-134.
Ueki T, Kaneda Y, Tsuitsui H, Nakanishi K, Sawa Y, Morishita R, Matsumoto K, et al. Hepatocyte growth factor gene therapy of liver cirrhosis in rats. Nat Med 1999; 5: 226-230.
Salgado S, Garcia J, Vera J, Siller F, Bueno M, Miranda A, Segura A, et al. Liver cirrhosis is reverted by urokinase-type plasminogen activator gene therapy. Molecular Therapy 2000; 2(6): 545-551.
Siller F, Salgado S, Sandoval A , Salazar A, Garcia J, Vera J, Galvan FJ, Juan Armendariz-Borunda. Treatment with human metalloproteinase-8 gene delivery ameliorates experimental rat liver cirrhosis. Gastroenterology. In press.