Hepatic gene therapy: Present and future
1993; Lippincott Williams & Wilkins; Volume: 18; Issue: 5 Linguagem: Inglês
10.1002/hep.1840180536
ISSN1527-3350
Autores Tópico(s)CRISPR and Genetic Engineering
ResumoHepatologyVolume 18, Issue 5 p. 1263-1273 Special ArticleFree Access Hepatic gene therapy: Present and future Fred D. Ledley M.D., Corresponding Author Fred D. Ledley M.D. Departments of Cell Biology and Pediatrics, Baylor College of Medicine, One Baylor Plaza, Section on Gastroenterology and Nutrition, Texas Children's Hospital, Houston, Texas 77030Department of Cell Biology, Baylor College of Medicine, One Baylor Plaza, Houston, TX 77030===Search for more papers by this author Fred D. Ledley M.D., Corresponding Author Fred D. Ledley M.D. Departments of Cell Biology and Pediatrics, Baylor College of Medicine, One Baylor Plaza, Section on Gastroenterology and Nutrition, Texas Children's Hospital, Houston, Texas 77030Department of Cell Biology, Baylor College of Medicine, One Baylor Plaza, Houston, TX 77030===Search for more papers by this author First published: November 1993 https://doi.org/10.1002/hep.1840180536Citations: 40AboutPDF ToolsRequest permissionExport citationAdd to favoritesTrack citation ShareShare Give accessShare full text accessShare full-text accessPlease review our Terms and Conditions of Use and check box below to share full-text version of article.I have read and accept the Wiley Online Library Terms and Conditions of UseShareable LinkUse the link below to share a full-text version of this article with your friends and colleagues. Learn more.Copy URL Share a linkShare onFacebookTwitterLinkedInRedditWechat References 1 Wilson JM, Grossman M, Raper SE, Baker JR Jr, Newton RS, Thoene JG. Ex vivo gene therapy of familial hypercholesterolemia. Hum Gene Ther 1992; 3: 179– 222. 2 Ledley FD, Woo SL, Ferry GD, Whisennand HH, Brandt ML, Darlington GJ, Demmler GJ, et al. Hepatocellular transplantation in acute hepatic failure and targeting genetic markers to hepatic cells. Hum Gene Ther 1991; 2: 331– 358. 3 Anderson WF. Human gene therapy. Science 1992; 256: 808– 813. 4 Miller AD. Human gene therapy comes of age. Nature 1992; 357: 455– 460. 5 Ledley FD. Are contemporary methods for somatic gene therapy suitable for clinical applicatioon? Clin Invest Med 1993; 16: 78– 88. 6 Rosenberg SA, Aebersold P, Cornetta K, Kasid A, Morgan RA, Moen R, Karson EM, et al. Gene transfer into humans: immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene trarosduction. N Engl J Med 1990; 323: 570– 578. 7 Rosenberg SA. Gene therapy for cancer. JAMA 1992; 268: 2416– 2419. 8 Miller AD. Retrovirus packaging cells. Hum Gene Ther 1990; 61: 5– 14. 9 Sratford-Perricaudet LD, Levrero M, Chasse JF, Perricaudet M, Briand P. Evaluation of the transfer and expression in mice of an enzyme-encoding gene using a human adenovirus vector. Hum Gene Ther 1990; 1: 241– 256. 10 Gilardi P, Courtney M, Pavirani A, Perricaudet M. Expression of human alpha 1-antitrypsin using a recombinant adenovirus vector. FEBS Lett 1990; 267: 60– 62. 11 Graham FL, Prevec L. Manipulation of adenovirus vectors. In: EJ Murray, ed. Methods in molecular biology. Vol 7: Gene transfer and expression protocols. Clifton, NJ: Humana Press, 1991: 109– 128. 12 Berkner KL. Development of adenovirus vectors for the expression of heterologous genes. Biotechniques 1988; 6: 616– 629. 13 Jaffe HA, Danel C, Longenecker G, Metzger M, Setoguchi Y, Rosenfeld MA, Gant TW, et al. Adenovirus-mediated in vivo gene transfer and expression in normal rat liver. Nat Genet 1992; 1: 372– 378. 14 Samulski RJ, Chang LS, Shenk T. Helper-free stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expression. J Virol 1989; 63: 3822– 3828. 15 Fink DJ, Sternberg LR, Weber PC, Mata M, Goins WF, Glorioso JC. In vivo expression of beta-galactosidase in hippocampal neurons by HSV-mediated gene transfer. Hum Gene Ther 1992; 3: 11– 19. 16 Geller AI, Keyomarsi K, Bryan J, Pardee AB. An efficient deletion mutant packaging system for defective herpes simplex virus vectors: potential applications to human gene therapy and neuronal physiology. Proc Natl Acad Sci USA 1990; 87: 8950– 8954. 17 Capecchi MR. High efficiency transformation by direct microinjection of DNA into cultured mammalian cells. Cell 1980; 22: 479– 488. 18 Wolff JA, Malone RW, Williams P, Chong W, Acsadi G, Jani A, Felgner PL. Direct gene transfer into mouse muscle in vivo. Science 1990; 247: 1465– 1468. 19 O'Malley BW Jr, Sikes ML, Adams RM, Finegold MJ, Ledley FD. Somatic gene therapy targeted to the thyroid: DNA mediated and viral mediated gene transfer in animal models. Transplant Proc 1992; 24: 2973– 2974. 20 Yang NS, Burkholder J, Roberts B, Martinell B, McCabe D. In vivo and in vitro gene transfer to mammalian somatic cells by particle bombardment. Proc Natl Acad Sci USA 1990; 87: 9568– 9572. 21 Felgner PL, Gadek TR, Holm M, Roman R, Chan HW, Wenz M, Northrup JP, et al. Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure. Proc Natl Acad Sci USA 1987; 84: 7413– 7417. 22 Wu GY, Wilson JM, Wu CH. Targeting genes: delivery and persistent expression of a foreign gene driven by mammalian regulatory elements in viva. J Biol Chem 1989; 264: 16985– 16987. 23 Wu GY, Wu CH. Receptor-mediated gene delivery and expression in vivo. J Biol Chem 1988; 263: 14621– 14624. 24 Wu GY, Wu CH. Evidence for targeted gene delivery to hepG2 hepatoma cells in vitro. Biochemistry 1988; 27: 887– 892. 25 Wu GY, Wu CH. Receptor-mediated in vitro gene transformation by a soluble DNA carrier system. J Biol Chem 1987; 262: 4429– 4432. 26 Wu GY, Wilson JM, Shalaby F, Grossman M, Shafritz DA, Wu CH. Receptor-mediated gene delivery in vivo: partial correction of genetic analbuminemia in Nagase rats. J Biol Chem 1991; 266: 14338– 14342. 27 Wilson JM, Wu CH, Wu GY. Targeting genes: delivery and persistent expression of a foreign gene driven by mammalian regulatory elements in vivo. J Biol Chem 1989; 264: 16985– 16987. 28 Wilson JM, Grossman M, Cabrera JA, Wu CH, Wu GY. A novel mechanism for achieving transgene persistence in vivo after somatic gene transfer into hepatocytes. J Biol Chem 1992; 267: 11483– 11489. 29 Wagner E, Zenke M, Cotten M, Beug H, Birnstiel ML. Transferrin-polycation conjugates as carriers for DNA uptake into cells. Proc Natl Acad Sci USA 1990; 87: 3410– 3414. 30 Wagner E, Plank C, Zatloukal K, Cotten M, Birnstiel ML. Influenza virus hemagglutinin HA-2 N-terminal fusogenic peptides augment gene transfer by transferrin-polylysine-DNA complexes: toward a synthetic virus-like gene-transfer vehicle. Proc Natl Acad Sci USA 1992; 89: 7934– 7938. 31 Wagner E, Zatloukal K, Cotten M, Kirlappos H, Mechtler K, Curiel DT, Birnstiel ML. Coupling of adenovirus to transferrinpolylysine/DNA complexes greatly enhances receptor-mediated gene delivery and expression of transfected genes. Proc Natl Acad Sci USA 1992; 89: 6099– 6103. 32 Anderson KD, Thompson JA, DiPietro JM, Montgomery KT, Reid LM, Anderson WF. Gene expression in implanted rat hepatocytes following retroviral-mediated gene transfer. Somat Cell Mol Genet 1989; 15: 215– 227. 33 Wilson JM, Johnston DE, Jefferson DM, Mulligan RC. Correction of the genetic defect in hepatocytes from the Watanabe heritable hyperlipidemic rabbit. Proc Natl Acad Sci USA. 1988; 85: 4421– 4425. 34 Roy Chowdhury J, Grossman M, Gupta SJ, Roy Chowdhury N, Baker JR Jr, Wilson JM. Long-term improvement of hypercholesterolemia after ex vivo gene therapy in LDLR-deficient rabbits. Science 1991; 254: 1802– 1805. 35 Kay MA, Baley P, Rothenberg S, Leland F, Fleming L, Ponder KP, Liu T, et al. Expression of human alpha1-antitrypsin in dogs after autologous transplantation of retroviral transduced hepatocytes. Proc Natl Acad Sci USA 1992; 89: 89– 93. 36 Ledley FD, Adams RM, Soriano HE, Darlington GJ, Finegold M, Lanford R, Carey D, et al. Development of a clinical protocol for hepatic gene transfer: lessons learned in pre-clinical studies. Pediatr Res 1993; 33: 313– 320. 37 Grossman M, Raper S, Wilson JM. Towards liver-directed gene therapy: retrovirus-mediated gene transfer into human hepatocytes. Somat Cell Mol Genet 1992; 17: 601– 607. 38 Adams RM, Soriano H, Wang M, Darlington G, Steffen D, Ledley FD. Transduction of primary human hepatocytes with amphotropic and xenotropic retroviral vectors. Proc Natl Acad Sci USA 1992; 89: 8981– 8985. 39 Grossman M, Raper SE, Wilson JM. Transplantation of genetically modified autologous hepatocytes into nonhuman primates: feasibility and short-term toxicity. Hum Gene Ther 1992; 5: 501– 510. 40 Ledley FD, Darlington GJ, Hahn T, Woo SL. Retroviral gene transfer into primary hepatocytes: implications for genetic therapy of liver specific functions. Proc Natl Acad Sci USA 1987; 84: 5335– 5339. 41 Wolff JA, Yee JK, Skelly HF, Moores JC, Respess JG, Friedmann Y, Leffert H. Expression of retrovirally transduced genes in primary cultures of adult rat hepatocytes. Proc Natl Acad Sci USA 1987; 84: 3344– 3348. 42 Wilson JM, Jefferson DM, Roy Chowdhury J, Novikoff PM, Johnston DE, Mulligan RC. Retrovirus-mediated transduction of adult hepatocytes. Proc Natl Acad Sci USA 1988; 85: 3014– 3018. 43 Wilson JM, Johnston DE, Jefferson DM, Mulligan RC. Correction of the genetic defect in hepatocytes from the Watanabe heritable hyperlipidemic rabbit. Proc Natl Acad Sci USA 1988; 85: 4421– 4425. 44 Wilson JM, Roy Chowdhury N, Grossman M, Wajsman R, Epstein A, Mulligan RC, Roy Chowdhury J. Temporary amelioration of hyperlipidemia in low density lipoprotein receptor deficient rabbits transplanted with genetically modified hepatocytes. Proc Natl Acad Sci USA 1990; 87: 8437– 8441. 45 Armentano D, Thompson AR, Darlington G, Woo SL. Expression of human factor IX in rabbit hepatocytes by retrovirus-mediated gene transfer: potential for gene therapy of hemophilia B. Proc Natl Acad Sci USA 1990; 87: 6141– 6145. 46 Soriano H, Brandt M, Adams RM, Gest A, Finegold M, Darlington G, Ledley FD. Retroviral mediated gene transfer and expression of marker genes in fetal ovine hepatocytes [Abstract]. Pediatr Res 1991; 29: 134A. 47 Cornetta K, Moen RC, Culver K, Morgan RA, McLachlin JR, Sturn S, Selegue J, et al. Amphotropic murine leukemia retrovirus is not an acute pathogen for primates. Hum Gene Ther 1990; 1: 15– 30. 48 Cornetta K, Morgan RA, Anderson WF. Safety issues related to retroviral mediated gene transfer in humans. Hum Gen Ther 1991; 2: 5– 14. 49 Moolten FL, Cupples LA. A model for predicting the risk of cancer consequent to retroviral gene therapy. Hum Gene Ther 1992; 5: 479– 486. 50 Belmont JW, MacGregor GR, Wagner-Smith K, Fletcher FA, Moore KA, Hawkins D, Villalon D, et al. Expression of human adenosine deaminase in murine hematopoietic cells. Mol Cel Biol 1986; 8: 5116– 5125. 51 Palmer TD, Rosman GJ, Osborne WR, Miller AD. Genetically modified skin fibroblasts persist long after transplantation but gradually inactivate introduced genes. Proc Natl Acad Sci USA 1991; 88: 1330– 1334. 52 Scharfmann R, Axelrod JH, Verma IM. Long-term in vivo expression of retrovirus-mediated gene transfer in mouse fibroblast implants. Proc Natl Acad Sci USA 1991; 88: 4626– 4630. 53 Peng H, Armentano D, Graham L, Ledley FD, Woo SLC. Retroviral-mediated gene transfer and expression of human phenylalanine hydroxylase in primary mouse hepatocytes. Proc Natl Acad Sci USA 1988; 85: 8146– 8150. 54 Bumgardner GL, Fasola C, Sutherland DE. Prospects for hepatocyte transplantation. HEPATOLOGY 1988; 8: 1158– 1161. 55 Fuller BJ. Transplantation of isolated hepatocytes. A review of current ideas. J Hepatol 1988; 7: 368– 376. 56 Gupta S, Roy Chowdhury J. Hepatocyte transplantation: back to the future. HEPATOLOGY 1992; 15: 156– 162. 57 Gupta S, Yerneni PR, Vemuru RP, Lee CD, Yellin EL, Bhargava KK. Studies on the safety of intrasplenic hepatocyte transplantation: relevance to ex vivo gene therapy and liver repopulation in acute hepatic failure. Hum Gene Ther 1993; 4: 249– 257. 58 Gupta S, Roy Chowdhury N, Jagtiani R, Gustin K, Aragona E, Shafritz DA, Roy Chowdhury J, et al. A novel system for transplantation of isolated hepatocytes utilizing HBsAg-producing transgenic donor cells. Transplantation 1990; 50: 472– 475. 59 Gupta S, Aragona E, Vemuru RP, Bhagarva KK, Burk RD, Roy Chowdhury J. Permanent engraftment and function of hepatocytes delivered to the liver: implications for gene therapy and liver repopulation. HEPATOLOGY 1990; 14: 144– 149. 60 Ponder KP, Gupta S, Leland F, Darlington G, Finegold M, DeMayo J, Ledley FD, et al. Mouse hepatocytes migrate to liver parenchyma and function indefinitely after intrasplenic transplantation. Proc Natl Acad Sci USA 1991; 88: 1217– 1221. 61 Ledley FD. Clinical application of somatic gene therapy in inborn errors of metabolism. J Inherit Metab Dis 1990; 13: 597– 616. 62 Hartman SC, Mulligan RC. Two dominant-acting selectable markers for gene transfer studies in mammalian cells. Proc Natl Acad Sci USA 1988; 85: 8047– 8051. 63 Lanford RE, Carey D, Estlack LE, Smith GC, Hay RV. Analysis of plasma protein and lipoprotein synthesis in long-term primary cultures of baboon hepatocytes maintained in serum-free medium. In Vitro Cell Dev Biol 1989; 25: 174– 182. 64 Sigal SH, Brill S, Fiorino AS, Reid LM. The liver as a stem cell and lineage system. Am J Physiol 1992; 263: 139– 148. 65 Gupta S, Lee CD, Aragona E, Burk RD, Yerneni PR. Hepatocyte transplantation based reconstitution of the liver for gene therapy [Abstract]. Clin Res 1993; 41: 158A. 66 Jaenisch R, Hoffmann E. Transcription of endogenous C-type viruses in resting and proliferating tissues of BALB/Mo mice. Virology 1979; 98: 289– 297. 67 Jaenisch R, Fan H, Croker B. Infection of preimplantation mouse embryos and of newborn mice with leukemia virus: tissue distribution of viral DNA and RNA and leukemogenesis in the adult animal. Proc Natl Acad Sci USA 1975; 72: 4008– 4012. 68 Albritton LM, Tseng L, Scadden D, Cunningham JM. A putative murine ecotropic retrovirus receptor gene encodes a multiple membrane-spanning protein and confers susceptibility to virus infection. Cell 1989; 57: 659– 666. 69 Albritton LM, Kim JW, Tseng L, Cunningham JM. Envelopebinding domain the cationic amino acid transporter determines the host range of ecotropic murine retroviruses. J Virol 1993; 67: 2091– 2096. 70 Kaleko M, Garcia JV, Miller AD. Persistent gene expression after retroviral gene transfer into liver cells in vivo. Hum Gene Ther 1991; 2: 27– 32. 71 Ferry N, Duplessis O, Houssin D, Danos O, Heard JM. Retroviral mediated gene transfer into hepatocytes in vivo. Proc Natl Acad Sci USA 1991; 88: 8377– 8381. 72 Kay MA, Li O, Liu TJ, Leland F, Toman C, Finegold M, Woo SL. Hepatic gene therapy: persistent expression of human alpha1-antitrypsin in mice after direct gene delivery in vivo. Hum Gene Ther, in press. 73 Cardoso JE, Branchereau S, Roy JP, Houssin D, Danos O, Heard JM. In situ retrovirus-mediated gene transfer into dog liver. Hum Gene Ther, in press. 74 Neda H, Wu CH, Wu GY. Chemical modification of an ecotropic murine leukemic virus results in redirection of its target cell specificity. J Biol Chem 1991; 266: 14143– 14146. 75 Welsh RM, Cooper NR, Jensen FC, Oldstone MB. Human serum lyses RNA tumor viruses. Nature 1975; 257: 612– 614. 76 Weiss R. The search for human RNA tumor viruses. In: R Weiss, N Teich, H Varmus, J Coffin, eds. RNA tumor viruses. Cold Spring Harbor: Cold Spring Harbor Laboratory, 1984; 1205– 1281. 77 Rosenfeld MA, Siegfried W, Yoshimura K, Yoneyama K, Fukayama M, Stier LE, Paakka PK, et al. Adenovirus-mediated transfer of a recombinant alpha 1-antitrypsin gene to the lung epithelium in vivo. Science 1991; 252: 431– 434. 78 Minutes of Recombinant DNA Advisory Committee meeting, December 1992. Washington, DC. 79 Stankovics J, Andrews E, Wu G, Ledley FD. Overexpression of human methylmalonyl CoA mutase (MCM) in mouse liver after in vivo gene delivery using asialoglycoprotein complexes [Abstract]. Am J Hum Genet 1992; 51: A177. 80 Haensler J, Szoka FC Jr. Synthesis and characterization of a trigalactosylated bisacridine compound to target DNA to hepatocytes. Bioconjug Chem 1993; 4: 85– 93. 81 Ledley FD. Pharmacokinetic issues in the use of genes as medicines. In J Wolff, ed. Gene therapy. New York: Birkhauser, 1993, in press. 82 Curiel DT, Agarwal S, Wagner E, Cotten M. Adenovirus enhancement of transferrin-polylysine-mediated gene delivery. Proc Natl Acad Sci USA 1991; 88: 8850– 8854. 83 Cristiano RJ, Smith LC, Woo SLC. Hepatic gene therapy: adenovirus enhancement of receptor-mediated gene delivery and expression in primary hepatocytes. Proc Natl Acad Sci USA 1993; 90: 2122– 2126. 84 Pearson CE, Frappier L, Zannis-Hadjopoulos M. Plasmids bearing mammalian DNA-replication origin-enriched (ors) fragments initiate semiconservative replication in a cell-free system. Biochim Biophys Acta 1991; 1090: 156– 166. 85 Bell D, Sabloff M, Zannis-Hadjopoulos M, Price G. Anticruciform DNA affinity purification of active mammalian origins of replication. Biochim Biophys Acta 1991; 1089: 299– 308. 86 Kato K, Kaneda Y, Sakurai M, Nakanishi M, Okada Y. Direct injection of hepatitis B virus DNA into liver induced hepatitis in adult rats. J Biol Chem 1991; 266: 22071– 22074. 87 Kato K, Nakanishi M, Kaneda Y, Uchida T, Okada Y. Expression of hepatitis B virus surface antigen in adult rat liver: cointroduction of DNA and nuclear protein by a simplified liposome method. J Biol Chem 1991; 266: 3361– 3364. 88 Leibiger I, Leibiger B, Sarrach D, Walther R, Zuhlke H. Genetic manipulation of rat hepatocytes in vivo: implications for a therapy model of type-1 diabetes. Biomed Biochim Acta 1990; 49: 1193– 1200. 89 Leibiger B, Leibiger I, Sarrach D, Zuhlke H. Expression of exogenous DNA in rat liver cells after liposome-mediated transfection in vivo. Biochem Biophys Res Commun 1991; 174: 1223– 1231. 90 Nicolau C, Cudd A. Liposomes as carriers of DNA. Crit Rev Ther Drug Carrier Syst 1989; 6: 239– 271. 91 Kaneda Y, Iwai K, Uchida T. Introduction and expression of the human insulin gene in adult rat liver. J Biol Chem 1989; 264: 12126– 12129. 92 Nicolau C, Legrand A, Grosse E. Liposomes as carriers for in vivo gene transfer and expression. Methods Enzymol 1987; 149: 157– 176. 93 Nandi PK, Legrand A, Nicolau C. Biologically active, recombinant DNA in clathrin-coated vesicles isolated from rat livers after in vivo injection of liposome-encapsulated DNA. J Biol Chem 1986; 261: 16722– 16726. 94 Spanjer HH, van Galen M, Roerdink FH, Regts J, Scherphof GL. Intrahepatic distribution of small unilamellar liposomes as a function of liposomal lipid composition. Biochim Biophys Acta 1986; 863: 224– 230. 95 Capecchi MR. Altering the genome by homologous recombination: homologous recombination review. Science 1989; 244: 1288– 1292. 96 Nirenberg M. Will society be prepared? Science 1967; 157: 633– 635. 97 Anderson WF, Fletcher JC. Gene therapy in human beings, when is it ethical to begin? N Engl J Med 1980; 303: 1293– 1297. 98 Friedmann TA, Roblin R. Gene therapy for human genetic disease. Science 1972; 175: 949– 952. 99 Nelson JR. The role of religions in the analysis of the ethical issues of human gene therapy. Hum Gene Ther 1991; 1: 43– 48. 100 Murray TH. Human gene therapy, the public, and the public policy. Hum Gene Ther 1990; 1: 49– 54. 101 Walters L. The ethics of human gene therapy. Nature 1986; 320: 225– 227. 102 Fletcher JC. Evolution of ethical debate about human gene therapy. Hum Gene Ther 1990; 1: 55– 68. 103 Office of Technology Assessment. Human gene therapy [background paper]. Washington, DC: U.S. Government Printing Office, 1984. 104 Surgeon General. President's commission for the study of ethical problems in medicine and biomedical and behavioral research. Splicing life. Washington, D.C.: U.S. Government Printing Office, 1983. 105 Friedmann T. Gene therapy, fact and fiction. Cold Spring Harbor: Cold Spring Harbor Laboratory, 1983. 106 Ledley FD. Clinical considerations in the design of protocols for somatic gene therapy. Hum Gene Ther 1991; 2: 77– 84. 107 Recombinant DNA Advisory Committee. Points to consider in the design and submission of human somatic cell gene therapy protocols. Federal Register 54: no 169, 36698– 36703 [Reprinted in Hum Gene Ther 1990; 1: 93– 103]. 108 Ledley FD, Brody B, Kozinetz C, Mize M. The challenge of follow-up for clinical trials of somatic gene therapy. Hum Gene Ther 1991; 3: 657– 664. 109 Wilson JM. Presentation to Recombinant DNA Advisory Committee, December 1992. Washington, DC. 110 Ledley FD. Somatic gene therapy in gastroenterology: approaches and applications. J Pediatr Gastroenterol Nutr 1992; 14: 328– 37. Citing Literature Volume18, Issue5November 1993Pages 1263-1273 ReferencesRelatedInformation
Referência(s)