AndinoR.2003. RNAi puts a lid on virus replication. Nat. Biotechnol., 21:629–630.
2.
AzumaH., PaulkN., RanadeA., DorrellC., Al-DhalimyM., EllisE., StromS., KayM.A., FinegoldM., GrompeM.2007. Robust expansion of human hepatocytes in Fah−/−/Rag2−/−/Il2rg−/− mice. Nat. Biotechnol., 25:903–910.
3.
BartoschB., DubuissonJ., CossetF.L.2003. Infectious hepatitis C virus pseudo-particles containing functional E1–E2 envelope protein complexes. J. Exp. Med., 197:633–642.
4.
BissigK.D., LeT.T., WoodsN.B., VermaI.M.2007. Repopulation of adult and neonatal mice with human hepatocytes: A chimeric animal model. Proc. Natl. Acad. Sci. U.S.A., 104:20507–20511.
5.
CaoT., MeulemanP., DesombereI., SällbergM., Leroux-RoelsG.2001. In vivo inhibition of anti-hepatitis B virus core antigen (HBcAg) immunoglobulin G production by HBcAg-specific CD4+ Th1-type T-cell clones in a hu-PBL-NOD/SCID mouse model. J. Virol., 75:11449–11456.
ChuahM.K., SchiednerG., ThorrezL., BrownB., JohnstonM., GillijnsV., HertelS., Van RooijenN., LillicrapD., CollenD., VandenDriesscheT., KochanekS.2003. Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors. Blood, 101:1734–1744.
8.
DandriM., BurdaM.R., TorokE., PollokJ.M., IwanskaA., SommerG., RogiersX., RoglerC.E., GuptaS., WillH., GretenH., PetersenJ.2001. Repopulation of mouse liver with human hepatocytes and in vivo infection with hepatitis B virus. Hepatology, 33:981–988.
9.
DavidoffA.M., GrayJ.T., NgC.Y., ZhangY., ZhouJ., SpenceY., BakarY., NathwaniA.C.2005. Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models. Mol. Ther., 11:875–888.
10.
EmotoK., TatenoC., HinoH., AmanoH., ImaokaY., AsahinaK., AsaharaT., YoshizatoK.2005. Efficient in vivo xenogeneic retroviral vector-mediated gene transduction into human hepatocytes. Hum. Gene Ther., 16:1168–1174.
11.
FollenziA., BattagliaM., LombardoA., AnnoniA., RoncaroloM.G., NaldiniL.2004. Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice. Blood, 103:3700–3709.
12.
GaoG.P., AlviraM.R., WangL., CalcedoR., JohnstonJ., WilsonJ.M.2002. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc. Natl. Acad. Sci. U.S.A., 99:11854–11859.
13.
GrimmD., LeeJ.S., WangL., DesaiT., AkacheB., StormT.A., KayM.A.2008. In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses. J. Virol., 82:5887–5911.
14.
GrompeM., al-DhalimyM., FinegoldM., OuC.N., BurlingameT., KennawayN.G., SorianoP.1993. Loss of fumarylacetoacetate hydrolase is responsible for the neonatal hepatic dysfunction phenotype of lethal albino mice. Genes Dev., 2298–2307.
15.
HsuM., ZhangJ., FlintM., LogvinoffC., Cheng-MayerC., RiceC.M., McKeatingJ.A.2003. Hepatitis C virus glycoproteins mediate pH-dependent cell entry of pseudotyped retroviral particles. Proc. Natl. Acad. Sci. U.S.A., 100:7271–7276.
16.
JiangH., LillicrapD., Patarroyo-WhiteS., LiuT., QianX., ScallanC.D., PowellS., KellerT., McMurrayM., LabelleA., NagyD., VargasJ.A., ZhouS., CoutoL.B., PierceG.F.2006. Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs. Blood, 108:107–115.
17.
KangY., XieL., TranD.T., SteinC.S., HickeyM., DavidsonB.L., McCrayP.B.Jr.2005. Persistent expression of factor VIII in vivo following nonprimate lentiviral gene transfer. Blood, 106:1552–1558.
KuboS., KataokaM., TatenoC., YoshizatoK., KawasakiY., KimuraT., Faure-KumarE., PalmerD.J., NgP., OkamuraH., KasaharaN.2009. In vivo stable transduction of humanized liver tissue in chimeric mice via high-capacity adenovirus–lentivirus hybrid vector. Hum. Gene Ther., 21:40–50.
20.
MannoC.S., PierceG.F., ArrudaV.R., GladerB., RagniM., RaskoJ., OzeloM.C., HootsK., BlattP., KonkleB., DakeM., KayeR., RazaviM., ZajkoA., ZehnderJ., NakaiH., ChewA., LeonardD., WrightJ.F., LessardR.R., SommerJ.M., TiggesM., SabatinoD., LukA., JiangH., MingozziF., CoutoL., ErtlH.C., HighK.A., KayM.A.2006. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat. Med., 12:342–347.
21.
MatraiJ., ChuahM.K., VandenDriesscheT.2009. Recent advances in lentiviral vector development and applications. Mol. Ther.in press.
22.
McCaffreyA.P., NakaiH., PandeyK., HuangZ., SalazarF.H., XuH., WielandS.F., MarionP.L., KayM.A.2003. Inhibition of hepatitis B virus in mice by RNA interference. Nat. Biotechnol., 21:639–644.
23.
MercerD.F., SchillerD.E., ElliottJ.F., DouglasD.N., HaoC., RinfretA., AddisonW.R., FischerK.P., ChurchillT.A., LakeyJ.R., TyrrellD.L., KnetemanN.M.2001. Hepatitis C virus replication in mice with chimeric human livers. Nat. Med., 7:927–933.
24.
MeulemanP., LibbrechtL., De VosR., de HemptinneB., GevaertK., VandekerckhoveJ., RoskamsT., Leroux-RoelsG.2005. Morphological and biochemical characterization of a human liver in a uPA-SCID mouse chimera. Hepatology, 41:847–856.
25.
NathwaniA.C., GrayJ.T., NgC.Y., ZhouJ., SpenceY., WaddingtonS.N., TuddenhamE.G., Kemball-CookG., McIntoshJ., Boon-SpijkerM., MertensK., DavidoffA.M.2006. Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver. Blood, 107:2653–2661.
OverturfK., Al-DhalimyM., TanguayR., BrantlyM., OuC.N., FinegoldM., GrompeM.1996. Hepatocytes corrected by gene therapy are selected in vivo in a murine model of hereditary tyrosinaemia type I. Nat. Genet., 12:266–273.
28.
PienG.C., Basner-TschakarjanE., HuiD.J., MentlikA.N., FinnJ.D., HasbrouckN.C., ZhouS., MurphyS.L., MausM.V., MingozziF., OrangeJ.S., HighK.A.2009. Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors. J. Clin. Invest., 119:1688–1695.
29.
PierceG.F., LillicrapD., PipeS.W., VandenDriesscheT.2007. Gene therapy, bioengineered clotting factors and novel technologies for hemophilia treatment. J. Thromb. Haemost., 5:901–906.
30.
RussellS.J.2003. Rise of the nanomachines. Nat. Biotechnol., 21:872–873.
31.
SandgrenE.P., PalmiterR.D., HeckelJ.L., DaughertyC.C., BrinsterR.L., DegenJ.L.1991. Complete hepatic regeneration after somatic deletion of an albumin-plasminogen activator transgene. Cell, 66:245–256.
32.
ShafritzD.A.2007. A human hepatocyte factory. Nat. Biotechnol., 25:871–872.
33.
TatenoC., YoshizaneY., SaitoN., KataokaM., UtohR., YamasakiC., TachibanaA., SoenoY., AsahinaK., HinoH., AsaharaT., YokoiT., FurukawaT., YoshizatoK.2004. Near completely humanized liver in mice shows human-type metabolic responses to drugs. Am. J. Pathol., 165:901–912.
34.
ThomasC.E., StormT.A., HuangZ., KayM.A.2004. Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adenoassociated virus vectors. J. Virol., 78:3110–3122.
35.
VandenDriesscheT., ThorrezL., NaldiniL., FollenziA., MoonsL., BernemanZ., CollenD., ChuahM.K.2002. Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo. Blood, 100:813–822.
36.
VandenDriesscheT., ThorrezL., Acosta-SanchezA., PetrusI., WangL., MaL., De WaeleL., IwasakiY., GillijnsV., WilsonJ.M., CollenD., ChuahM.K.2007. Efficacy and safety of adeno-associated viral vectors based on serotype 8 and 9 versus lentiviral vectors for hemophilia B gene therapy. J. Thromb. Haemost., 5:16–24.
37.
XuD., AlipioZ., FinkL.M., AdcockD.M., YangJ., WardD.C., MaY.2009. Phenotypic correction of murine hemophilia A using an iPS cell-based therapy. Proc. Natl. Acad. Sci. U.S.A., 106:808–813.
38.
YamadaT., IwasakiY., TadaH., IwabukiH., ChuahM.K., VandenDriesscheT., FukudaH., KondoA., UedaM., SenoM., TanizawaK., KurodaS.2003. Nanoparticles for the delivery of genes and drugs to human hepatocytes. Nat. Biotechnol., 21:885–890.
