• 1
    Kay MA, Glorioso JC, Naldini L. Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics. Nat Med 2001; 7:3340.
  • 2
    Steer CJ, Kren BT, Roy Chowdhury N, Roy Chowdhury J. Liver-directed gene therapy. In: AriasIM, BoyerJL, ChisariFV, FaustoN, SchachterD, ShafritzDA, eds. The Liver: Biology and Pathobiology. 4th ed. Philadelphia: Lippincott Williams and Wilkins, 2001:941962.
  • 3
    Naldini L, Blomer U, Gallay P, Ory D, Mulligan R, Gage FH, Verma IM, et al. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 1996; 272:263267.
  • 4
    Emerman M. Learning from lentiviruses. Nat Genet 2000; 24:89.
  • 5
    Naldini L, Blomer U, Gage FH, Trono D, Verma IM. Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector. Proc Natl Acad Sci U S A 1996; 93:1138211388.
  • 6
    Gusella GL, Fedorova E, Hanss B, Marras D, Klotman ME, Klotman PE. Lentiviral gene transduction of kidney. Hum Gene Ther 2002; 13:407414.
  • 7
    Kafri T, Blomer U, Peterson DA, Gage FH, Verma IM. Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors. Nat Genet 1997; 17:314317.
  • 8
    Park F, Ohashi K, Chiu W, Naldini L, Kay MA. Efficient lentiviral transduction of liver requires cell cycling in vivo. Nat Genet 2000; 24:4952.
  • 9
    Pfeifer A, Kessler T, Yang M, Baranov E, Kootstra N, Cheresh DA, Hoffman RM, et al. Transduction of liver cells by lentiviral vectors: analysis in living animals by fluorescence imaging. Mol Ther 2001; 3:319322.
  • 10
    Follenzi A, Sabatino G, Lombardo A, Boccaccio C, Naldini L. Efficient gene delivery and targeted expression to hepatocytes in vivo by improved lentiviral vectors. Hum Gene Ther 2002; 13:243260.
  • 11
    Vanden Driessche T, Thorrez L, Maldini L, Follenzi A, Moons L, Berneman Z, Collen D, et al. Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo. Blood 2002; 100:813822.
  • 12
    Stinski MF, Roehr TJ. Activation of the major immediate early gene of human cytomegalovirus by cis-acting elements in the promoter-regulatory sequence and by virus-specific trans-acting components. J Virol 1985; 55:431441.
  • 13
    Kay MA, Baley P, Rothenberg S, Leland F, Fleming L, Ponder KP, Liu T, et al. Expression of human α 1-antitrypsin in dogs after autologous transplantation of retroviral transduced hepatocytes. Proc Natl Acad Sci U S A 1992; 89:8993.
  • 14
    Kay MA, Li Q, Liu TJ, Leland F, Toman C, Finegold M, Woo SL. Hepatic gene therapy: persistent expression of human α 1-antitrypsin in mice after direct gene delivery in vivo. Hum Gene Ther 1992; 3:641647.
  • 15
    Michalopoulos GK, DeFrances MC. Liver regeneration. Science 1997; 276:6066.
  • 16
    Shafritz DA, Dabeva MD. Liver stem cells and model systems for liver repopulation. J Hepatol 2002; 36:552564.
  • 17
    Rhim J, Sangren EP, Degan JL, Palmiter RD, Brinster RL. Replacement of diseased mouse liver by hepatic cell transplantation. Science 1994; 263:11491152.
  • 18
    Overturf K, Al-Dhalimy M, Tanguay R, Brantly M, Ou CN, Finegold M, Grompe M. Hepatocytes corrected by gene therapy are selected in vivo in a murine model of hereditary tyrosinaemia type I. Nat Genet 1996; 12:266273.
  • 19
    Mignon A, Guidotti JE, Mitchell C, Fabre M, Wernet A, De La Coste A, Soubrane O, et al. Selective repopulation of normal mouse liver by Fas/CD95-resistant hepatocytes. Nat Med 1998; 4:11851188.
  • 20
    Laconi E, Oren R, Mukhopadhyay DK, Hurston E, Laconi S, Pani P, Dabeva MD, et al. Long-term, near-total liver replacement by transplantation of isolated hepatocytes in rats treated with retrorsine. Am J Pathol 1998; 153:319329.
  • 21
    Guha C, Sharma A, Gupta S, Alfieri A, Gorla GR, Gagandeep S, Sokhi R, et al. Amelioration of radiation-induced liver damage in partially hepatectomized rats by hepatocyte transplantation. Cancer Res 1999; 59:58715874.
  • 22
    Malhi H, Gorla GR, Irani AN, Annamaneni P, Gupta S. Cell transplantation after oxidative hepatic preconditioning with radiation and ischemia-reperfusion leads to extensive liver repopulation. Proc Natl Acad Sci U S A 2002; 99:1311413119.
  • 23
    Sandhu JS, Petkov PM, Dabeva MD, Shafritz DA. Stem cell properties and repopulation of the rat liver by fetal liver epithelial progenitor cells. Am J Pathol 2001; 159:13231334.
  • 24
    Donello JE, Trono D, Hope TJ. Woodchuck hepatitis posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors. J Virol 1999; 73:28862892.
  • 25
    Seglen PO. Preparation of isolated rat liver cells. Methods Cell Biol 1976; 13:2983.
  • 26
    Kreamer BL, Staecker JL, Sawada N, Sattler GL, Hsia MT, Pitot HC. Use of a low-speed, iso-density percoll centrifugation method to increase the viability of isolated rat hepatocyte preparations. In Vitro Cell Dev Biol 1986; 22:201211.
  • 27
    Block GD, Locker J, Bowen WC, Petersen BE, Katyal S, Strom SC, Riley T, et al. Population expansion, clonal growth, and specific differentiation patterns in primary cultures of hepatocytes induced by HGF/SF, EGF, and TGF α in a chemically defined (HGM) medium. J Cell Biol 1996; 132:11331149.
  • 28
    Dabeva MD, Petkov PM, Sandhu J, Oren R, Laconi E, Hurston E, Shafritz DA. Proliferation and differentiation of fetal liver epithelial progenitor cells after transplantation into adult rat liver. Am J Pathol 2000; 156:20172031.
  • 29
    Dabeva MD, Hwang SG, Vasa SR, Hurston E, Novikoff PM, Hixson DC, Gupta S, et al. Differentiation of pancreatic epithelial progenitor cells into hepatocytes following transplantation into rat liver. Proc Natl Acad Sci U S A 1997; 94:73567361.
  • 30
    Nguyen T, Oberholzer J, Birraux J, Majno P, Morel P, Trono D. Highly efficient lentiviral vector-mediated transduction of nondividing, fully reimplantable primary hepatocytes. Mol Ther 2002; 6:199209.
  • 31
    Ohashi K, Park F, Kay MA. Role of hepatocyte direct hyperplasia in lentivirus: mediated liver transduction in vivo. Human Gene Ther 2002; 13:653663.
  • 32
    Baskar JF, Smith PP, Nilaver G, Jupp RA, Hoffmann S, Peffer NJ, Tenney DJ, et al. The enhancer domain of the human cytomegalovirus major immediate-early promoter determines cell type-specific expression in transgenic mice. J Virol 1996; 70:32073214.
  • 33
    Pinkert CA, Ornitz DM, Brinster RL, Palmiter RD. An albumin enhancer located 10 kb upstream functions along with its promoter to direct efficient, liver-specific expression in transgenic mice. Genes Dev 1987; 1:268276.
  • 34
    Murakami H, Sanderson ND, Nagy P, Marino PA, Merlino G, Thorgeirsson SS. Transgenic mouse model for synergistic effects of nuclear oncogenes and growth factors in tumorigenesis: interaction of c-myc and transforming growth factor α in hepatic oncogenesis. Cancer Res 1993; 53:17191723.
  • 35
    Conner EA, Lemmer ER, Omori M, Wirth PJ, Factor VM, Thorgeirsson SS. Dual functions of E2F-1 in a transgenic mouse model of liver carcinogenesis. Oncogene 2000; 19:50545062.
  • 36
    Sund NJ, Ang SL, Sackett SD, Shen W, Daigle N, Magnuson MA, Kaestner KH. Hepatocyte nuclear factor 3β (Foxa2) is dispensable for maintaining the differentiated state of the adult hepatocyte. Mol Cell Biol 2000; 20:51755183.
  • 37
    Follenzi A, Ailles LE, Bakovic S, Geuna M, Naldini L. Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences. Nat Genet 2000; 17:217222.
  • 38
    Overturf K, Al-Dhalimy M, Manning K, Ou CN, Finegold M, Grompe M. Ex vivo hepatic gene therapy of a mouse model of hereditary tyrosinemia type I. Hum Gene Ther 1998; 9:295304.
  • 39
    Guidotti J-E, Mallet VO, Mitchell C, Fabre M, Schoevaert D, Opolon P, Parlier D, et al. Selection of in vivo retrovirally transduced hepatocytes leads to efficient and predictable mouse liver repopulation. FASEB J 2001; 15:18491851.