Selected Leboulch Lab Publications

Raich N, Mignotte V, Dubart A, Beaupain D, Leboulch P, Romana M, Chabret C, Charnay P, Papayannopoulos T, Goossens M, Romeo PH.  Regulated expression of the overlapping ubiquitous and erythroid transcription units of the human porphobilinogen deaminase (PBG-D) gene introduced into non erythroid and erythroid cells.  J Biol Chem. 1989; 264:10186-92.  PubMed Link

Castella-Escola J, Ojcius DM, Leboulch P, Joulin V, Blouquit Y, Garel MC, Valentin C, Rosa R, Climent-Romeo F, Cohen-Solal M.  Isolation and characterization of the gene encoding the muscle-specific isozyme of human phosphoglycerate mutase.  Gene 1990; 91:225-32.  PubMed Link

Leboulch P, Huang GM, Humphries RK, Oh YH, Eaves CJ, Tuan DY, London IM.  Mutagenesis of retroviral vectors transducing human beta-globin gene and beta-globin locus control region derivatives results in stable transmission of an active transcriptional structure. EMBO J. 1994; 13:3065-76.  PubMed Link

Takekoshi KJ, Oh YH, Westerman KA, London IM, Leboulch P.  Retroviral transfer of a beta-globin / delta-globin hybrid gene aimed at the gene therapy of sickle cell disease.  Proc Natl Acad Sci USA 1995; 92: 3014-8.  PubMed Link

Rosenwald IB, Kaspar R, Rousseau D, Gehrke L, Leboulch P, Chen JJ, Schmidt EV, Sonenberg N, London IM.  Eukaryotic translation initiation factor 4E regulates expression of cyclin D1 at transcriptional and post-transcriptional levels.  J Biol Chem 1995; 270:21176-80.  PubMed Link

Westerman KA, Leboulch P.  Reversible cell immortalization mediated by retroviral transfer and site-specific recombination.  Proc Natl Acad Sci USA 1996; 93:8971-76.  PubMed Link

Liger D, vanderSpek JC, Gaillard C, Cansiera C, Murphy JR, Leboulch P, Gillet D. Characterization and receptor specific toxicity of two diphtheria toxin-related interleukin-3 fusion proteins DAB389-mIL-3 and DAB389-(Gly4Ser)2-mIL-3. FEBS Lett 1997; 406:157-61.  PubMed Link

Qing K, Bachelot T, Mukherjee P, Peng L, Wang X-S, Yoder MC, Leboulch P, Srivastava A.  Adeno-associated virus 2-mediated transfer of ecotropic retrovirus receptor gene augments retroviral transduction of established and primary human cells.  J Virol 1997; 71:5663-67.  PubMed Link

Bouhassira EE, Westerman K, Leboulch P.  Transcriptional behavior of LCR enhancer elements integrated at the same chromosomal locus by recombinase-mediated cassette exchange.  Blood 1997; 90:3332-44.  PubMed Link

Raftopoulos H, Ward M, Leboulch P, Bank A.  Long-term transfer and expression of the human beta-globin gene in a mouse transplant model.  Blood 1997; 90:3414-22.  PubMed Link

Pawliuk R, Bachelot T, Raftopoulos H, Kalberer C, Humphries RK, Bank A, Leboulch P.  Retroviral vectors aimed at the gene therapy of human beta-globin gene disorders.  Ann N Y Acad Sci 1998; 850:151-62.  PubMed Link

Hennemann, B, Conneally, E, Pawliuk, R, Leboulch, P, Rose-John, S, Reid, D, Chuo, JY, Humphries, RK, Eaves, CJ.  Optimization of retroviral-mediated gene transfer to human NOD/SCID mouse repopulating cord blood cells through a systematic analysis of protocol variables.  Exp Hematol 1999; 27:817-25.  PubMed Link

Feng YQ, Seibler J, Alami R, Eisen A, Westerman KA, Leboulch P, Fiering S, Bouhassira, EE.  Site-Specific Chromosomal Integration In Mammalian cells:  Highly Efficient CRE Recombinase-Mediated Cassette Exchange. J Mol Biol 1999; 292:779-85  PubMed Link

Lai CC, Gouras P, Doi K, Lu F, Kjeldbye H, Goff SP, Pawliuk R, Leboulch P, Tsang SH.  Tracking RPE transplants labeled by retroviral gene transfer with green fluorescent protein.  Invest Ophtalmol Vis Sci 1999; 40:2141-46.  PubMed Link

Pawliuk R, Bachelot T, J. Wise RJ, Mathews-Roth MM, Leboulch P.  Long-term cure of the photosensitivity of murine erythropoietic protoporphyria by preselective gene therapy.  Nat Med 1999; 5:768-73.  PubMed Link

Rubin J, Pasceri P, Wu X, Leboulch P, Ellis J. Locus Control Region Activity by 5’HS3 Requires a Functional Interaction With beta-Globin Gene Regulatory Elements:  Expression of Novel beta/gamma-Globin Hybrid Transgenes.  Blood 2000; 95:3242-49.  PubMed Link

Kalberer CP, Pawliuk R, Imren S, Bachelot T, Takekoshi KJ, Fabry M, Eaves CJ, London IM, Humphries RK, Leboulch P. Preselection of Retrovirally Transduced Bone Marrow Avoids Subsequent Stem Cell Gene Silencing and Age-Dependent Extinction of Expression of Human beta-Globin in Engrafted Mice. Proc Natl Sci USA 2000; 97:5411-15.  PubMed Link

Kobayashi N, Noguchi H, watanabe T, Matsumura T, Totsugawa T, Fujiwara T, Westerman K, Leboulch P, Fox IJ, Tanaka N. Establishment of a tightly regulated human cell line for the development of hepatocyte transplantation. Hum Cell 2000; 13:7-13.  PubMed Link

Crosby JS, Chefalo PJ, Yeh I, Ying S, London IM, Leboulch P, Chen JJ. Regulation of hemoglobin synthesis and proliferation of diffrerentiating erythroid cells by heme-regulated eIF-2alpha kinase. Blood 2000; 96:3241-48.  PubMed Link

Kobayashi N, Fujiwara T, Westerman KA, Inoue Y, Sakaguchi M, Noguchi H, Miyazaki M, Tanaka N, Fox IJ, Leboulch P.  Prevention of Acute Liver Failure in Mice with Reversibly Immortalized Human Hepatocytes. Science 2000; 287:1258-62.  PubMed Link

Cai J, Ito M, Westerman KA, Kobayashi N, Leboulch P, Fox IJ. Construction of a non-tumorigenic rat hepatocyte cell line for transplantation: reversal of hepatocyte immortalization by site-specific excision of the SV40 T antigen. J Hepatology 2000; 33:701-8.  PubMed Link

Kobayashi N, Noguchi H, Fujiwara T, Westerman KA, Leboulch P, Tanaka N.  Establishment of a highly differentiated immortalized adult human hepatocyte cell line by retroviral gene transfer.  Transplant Proc 2000; 32:2368-69.  PubMed Link

Kobayashi N, Noguchi H, Westerman KA, Matsumura T, Watanabe T, Totsugawa T, Fujiwara T, Leboulch P, Tanaka N. Efficient Cre/LoxP site-specific recombination in a HepG2 human liver cell line.  Cell Transplant 2000; 9:737-42.  PubMed Link

Kobayashi N, Noguchi H, Watanabe T, Matsumura T, Totsugawa T, Fujiwara T, Taguchi T, Urata H, Kishimoto N, Hayashi N, Nakaji S, Westerman KA, Leboulch P, Murakami T, Tanaka N. A new approach to develop a biohybrid artificial liver using a tightly regulated human hepatocyte cell line. Hum Cell 2000; 13:229-35.  PubMed Link

Kobayashi N, Noguchi H, Watanabe T, Matsumura T, Totsugawa T, Fujiwara T, Westerman KA, Leboulch P, Fox IJ, Tanaka N. Establishment of a tightly regulated human cell line for the development of hepatocyte transplantation. Hum Cell 2000; 13:7-13.  PubMed Link

Kobayashi N, Miyazaki M, Westerman KA, Noguchi H, Sakaguchi M, Totsugawa T, Watanabe T, Matsumura T, Fujiwara T, Leboulch P, Tanaka N, Namba M. Construction of a differentiated human hepatocyte cell line expressing the herpes simplex virus-thymidine kinase gene. ASAIO J 2001; 47:476-80.  PubMed Link

Kobayashi N, Westerman KA, Taguchi T, Sakaguchi M, Fujiwara T, Urata H, Kishimoto N, Hayashi N, Nakaji S, Murakami T, Leboulch P, Tanaka N. Expansion of human hepatocyte populations by a retroviral gene transfer of simian virus 40 large T antigen. ASAIO J 2001; 47:481-5.  PubMed Link

Kobayashi N, Noguchi H, Westerman KA, Watanabe T, Matsumura T, Totsugawa T, Fujiwara T, Leboulch P, Tanaka N. Successful retroviral gene transfer of simian virus 40 T antigen and herpes simplex virus-thymidine kinase into human hepatocytes. Cell Transplant 2001; 10:377-81.  PubMed Link

Kobayashi N, Noguchi H, Westerman KA, Watanabe T, Matsumura T, Totsugawa T, Fujiwara T, Leboulch P, Tanaka N. Cre/loxP-based reversible immortalization of human hepatocytes. Cell Transplant 2001; 10:383-6.  PubMed Link

Jouanneau E, Alberti L, Nejjari M, Treilleux I, Vilgrain I, Duc A, Combaret V, Favrot M, Leboulch P, Bachelot T. Lack of antitumor activity of recombinant endostatin in a human neuroblastoma xenograft model. J Neurooncol 2001; 51: 11-8.  PubMed Link  

Kobayashi N, Westerman KA, Tanaka N, Fox IJ, Leboulch P. A rreversibly immortalized human hepatocyte cell line as a source of hepatocyte-based biological support. Addict Biol 2001; 6:293-300.  PubMed Link

Han A-P, Yu C, Lu L, Fujiwara Y, Browne C, Chin G, Fleming M, Leboulch P, Orkin SH, Chen J-J. HRI is required for translational regulation and survival of erythroid precursors in iron deficiency. EMBO J 2001; 20:1-10.  PubMed Link

Pawliuk R, Westerman KA, Fabry ME, Payen E, Tighe R, Bouhassira EE, Acharya SA, Ellis J, London IM, Eaves CJ, Humphries RK, Beuzard Y, Nagel RL, Leboulch P. Correction of sickle cell disease in transgenic mouse models by gene therapy. Science 2001; 294:2368-71.  PubMed Link

Noguchi H, Kobayashi N, Westerman KA, Sakaguchi M, Okitsu T, Totsugawa T, Watanabe T, Matsumura T, Fujiwara T, Ueda T, Miyazaki M, Tanaka N, Leboulch P. Controlled expansion of human endothelial cell population by Cre/LoxP-based reversible immortalization. Hum Gene Ther 2002; 13:321-34.  PubMed Link

Pawliuk R Bachelot T, Zurkiya O, Eriksson A, Cao Y, Leboulch P. Continuous intravascular secretion of endostatin in mice from transduced hematopoietic stem cells. Mol Ther 2002; 5:345-51.  PubMed Link

Eisterer W, Jiang X, Bachelot T, Pawliuk R, Abramovich C, Leboulch P, Hogge D, Eaves CJ. Unfulfilled promise of endostatin in a gene therapy-xenotransplant model of human acute lymphocytic leukemia. Mol Ther 2002; 5:352-59.  PubMed Link

Allain JE, Dagher I,  Mahieu-Caputo D, Loux N, Andreoletti M, Westerman KA, Briand P, Franco D, Leboulch P *, Weber A. Immortalization of the primate bipotent epithelial liver progenitor cell. Proc Natl Sci USA 2002; 99:3639-44 (* co-senior author).  PubMed Link

Gouze E, Pawliuk R, Pilapil C, Gouze J-N, Fleet C, Palmer GD, Evans CH, Leboulch P*, Ghivizzani SC. In vivo gene delivery to synovium by lentiviral vectors. Mol Ther 2002 (* co-senior author).  PubMed Link

Eriksson A, Cao R, Pawliuk R, Berg S-M, Fleet C, Tritsaris K, Dissing S, Leboulch P, Cao Y. Placental growth factor antagonizes VEGF-Induced Angiogenesis and tumor growth by the formation of functionally inactive PLGF-1/VEGF heterodimers. Cancer Cell 2002; 1:99-108.  PubMed Link

Maurisse R, Feugeas JP, Biet E, Kuzniak I, Leboulch P, Dutreix M, Sun JS. A new method (GOREC) for direct mutagenesis and gene repair by homologous recombination. Gene Ther 2002; 9:703-7.  PubMed Link

Imren S, Payen E, Westerman KA, Pawliuk R, Fabry ME, Eaves CJ, Cavilla B, Wadsworth LD, Beuzard Y, Bouhassira EE, Russell R, London IM, Nagel RL, Leboulch P *, Humphries RK*. Permanent and panerythroid correction of murine beta thalassemia by multiple lentiviral integration in hematopoietic stem cells. Proc Natl Sci USA (2002); 99:14380-5. (*Co-senior authors).  PubMed Link

Nicolini FE, Imren S, Oh IH, Humphries RK, Leboulch P, Fabry ME, Nagel RL, Eaves CJ. Expression of a human beta-globin transgene in erythroid cells derived from retrovirally transduced transplantable human fetal liver and cord blood cells. Blood. (2002); 100:1257-64.  PubMed Link

Cai J, Ito M, Nagata H, Westerman KA, Lafleur D, Chowdhury JR, Leboulch P, Fox IJ. Treatment of liver failure in rats with end-stage cirrhosis by transplantation of immortalized hepatocytes. Hepatol. (2002); 36:386-94.  PubMed Link

Gouze E, Pawliuk R, Gouze JN, Pilapil C, Fleet C, Palmer GD, Evans CH, Leboulch P*, Ghivizzani SC*. Lentiviral-mediated gene delivery to synovium: potent intra-articular expression with amplification by inflammation. Mol Ther 2003; 7:460-6.  PubMed Link

Cao R, Brakenhielm E, Pawliuk R, Wariaro D, Post M, Wahlberg E, Leboulch P, Cao Y. Angiogenic synergism, vascular stability and improvement of hind limb ischemia by a combination of PDGF-BB and FGF-2. Nat Med 2003; 9:604-13.  PubMed Link

de Franceschi L, Baron A, Scarpa A, Adrie C, Janin A, Barbi S, Kister J, Rouyer-Fessard P, Corrocher R, Leboulch P, Beuzard Y. Inhaled nitric oxide protects transgenic SAD mice from sickle cell disease-specific lung injury induced by hypoxia/reoxygenation. Blood 2003; 102:1087-96.  PubMed Link

Nishioka K, Fujimori Y, Hashimoto-Tamaoki T, Kai S, Qiu H, Kobayashi N, Tanaka N, Westerman KA, Leboulch P, Hara H. Immortalization of bone marrow-derived human mesenchymal stem cells by removable simian virus 40T antigen gene: analysis of the ability to support expansion of cord blood hematopoietic progenitor cells. Int J Oncol 2003; 23:925-32.  PubMed Link

Watanabe T, Shibata N, Westerman KA, Okitsu T, Allain JE, Sakaguchi M, Totsugawa T, Maruyama M, Matsumura T, Noguchi H, Yamamoto S, Hikida M, Ohmori A, Reth M, Weber A, Tanaka N, Leboulch P, Kobayashi N. Establishment of immortalized human hepatic stellate scavenger cells to develop bioartificial livers. Transplantation 2003; 75:1873-80.  PubMed Link

Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P, Lim A, Osborne CS, Pawliuk R, Morillon E, Sorensen R, Forster A, Fraser P, Cohen JI, de Saint Basile G, Alexander I, Wintergerst U, Frebourg T, Aurias A, Stoppa-Lyonnet D, Romana S, Radford-Weiss I, Gross F, Valensi F, Delabesse E, Macintyre E, Sigaux F, Soulier J, Leiva LE, Wissler M, Prinz C, Rabbitts TH, Le Deist F, Fischer A, Cavazzana-Calvo M. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003; 302(5644):415-9.  PubMed Link

Oh IH, Fabry ME, Humphries RK, Pawliuk R, Leboulch P, Hoffman R, Nagel RL, Eaves C. Expression of an anti-sickling beta-globin in human erythroblasts derived from retrovirally transduced primitive normal and sickle cell disease hematopoietic cells. Exp Hematol 2004; 32:461-9.  PubMed Link

Rivier F, Alkan O, Flint AF, Muskiewicz K, Allen PD, Leboulch P, Gussoni E.Role of bone marrow cell trafficking in replenishing skeletal muscle SP and MP cell populations. J Cell Sci 2004; 117:1979-88.  PubMed Link

Geromin D, Bourge JF, Soulie A, Pawliuk R, Fleet C, Michel E, Denizot Y, Berthou C, Leboulch P, Sigaux F, Sasportes M. Glycoprotein 170 induces platelet-activating factor receptor membrane expression and confers tumor cell hypersensitivity to NK-dependent cell lysis. J Immunol 2004; 172:3604-11.  PubMed Link

Maruyama M, Kobayashi N, Westerman KA, Sakaguchi M, Allain JE, Totsugawa T, Okitsu T, Fukazawa T, Weber A, Stolz DB, Leboulch P, Tanaka N. Establishment of a highly differentiated immortalized human cholangiocyte cell line with SV40T and hTERT. Transplantation 2004; 77:446-51.  PubMed Link

Okitsu T, Kobayashi N, Jun HS, Shin S, Kim SJ, Han J, Kwon H, Sakaguchi M, Totsugawa T, Kohara M, Westerman KA, Tanaka N, Leboulch P, Yoon JW. Transplantation of reversibly immortalized insulin-secreting human hepatocytes controls diabetes in pancreatectomized pigs. Diabetes 2004; 53:105-12.  PubMed Link

Matsumura T, Takesue M, Westerman KA, Okitsu T, Sakaguchi M, Fukazawa T, Totsugawa T, Noguchi H, Yamamoto S, Stolz DB, Tanaka N, Leboulch P, Kobayashi N. Establishment of an immortalized human-liver endothelial cell line with SV40T and hTERT. Transplantation 2004; 77:1357-65.  PubMed Link

Mahieu-Caputo D, Allain JE, Branger J, Coulomb A, Delgado JP, Andreoletti M, Mainot S, Frydman R, Leboulch P, Di Santo JP, Capron F, Weber A. Repopulation of athymic mouse liver by cryopreserved early human fetal hepatoblasts. Hum Gene Ther 2004; 15:1219-28.  PubMed Link

de Piedoue G, Maurisse R, Kuzniak I, Lopez B, Perrin A, Negre O, Leboulch P, Feugeas JP. Improving gene replacement by intracellular formation of linear homologous DNA.  J Gene Med 2005; 7:649-56.  PubMed Link

Imren S, Fabry ME, Westerman KA, Pawliuk R,  Tang P,  Rosten PM, Nagel RL, Leboulch P,  Eaves CJ, Humphries RK. High-level beta-globin expression and preferred intragenic integration after lentiviral transduction of human cord blood stem cells. J Clin Invest 2004; 114:953-62.  PubMed Link

Pawliuk R, Tighe R, Wise RJ, Mathews-Roth MM, Leboulch P. Prevention of murine erythropoietic protoporphyria-associated skin photosensitivity and liver disease by dermal and hepatic ferrochelatase. J Invest Dermatol 2005; 124:256-62.  PubMed Link

Dalle B, Rubin JE, Alkan O, Sukonnik T, Pasceri P, Yao S, Pawliuk R, Leboulch P, Ellis J. eGFP reporter genes silence LCRbeta-globin transgene expression via CpG dinucleotides. Mol Ther 2005; 11:591-9.  PubMed Link

Zhang JL, Cai J, Walls S, Jackson JD, Kuszynski CA, Zhao Y, Pawliuk R, Leboulch P, Fox IJ. Tolerance by selective in vivo expansion of foreign major histocompatibility complex-transduced autologous bone marrow. Transplantation 2005; 80:362-9.  PubMed Link

Kadri Z, Maouche-Chretien L, Rooke H, Orkin S, Romeo P-H, Mayeux P, Leboulch P *,  Chretien S*. PI3K/AKT induced by erythropoietin renders the erythroid differentiation factor GATA-1 competent for TIMP-1 gene transactivation. Mol Cell Biol 2005; 25:7412-22.  PubMed Link

Narushima M, Kobayashi N, Okitsu T, Tanaka Y, Li SA, Chen Y, Miki A, Tanaka K, Nakaji S, Takei K, Gutierrez AS, Rivas-Carrillo JD, Navarro-Alvarez N, Jun HS, Westerman KA, Noguchi H, Lakey JR, Leboulch P, Tanaka N, Yoon JW. A human beta-cell line for transplantation therapy to control type 1 diabetes. Nat Biotechnol 2005; 23:1274-82. (On-the-cover)  PubMed Link

Bank A, Dorazio R, Leboulch P. A Phase I/II Clinical Trial of {beta}-Globin Gene Therapy for Beta-Thalassemia. Ann N Y Acad Sci 2005; 1054:308-16.  PubMed Link

Qiu HY, Fujimori Y, Nishioka K, Yamaguchi N, Hashimoto-Tamaoki T, Sugihara A, Terada N, Nagaya N, Kanda M, Kobayashi N, Tanaka N, Westerman KA, Leboulch P, Hara H. Postnatal neovascularization by endothelial progenitor cells immortalized with the simian virus 40T antigen gene. Int J Oncol 2006; 28:815-21.  PubMed Link

Totsugawa T, Yong C, Rivas-Carrillo JD, Soto-Gutierrez A, Navarro-Alvarez N, Noguchi H, Okitsu T, Westerman KA, Kohara M, Reth M, Tanaka N, Leboulch P, Kobayashi N. Survival of liver failure pigs by transplantation of reversibly immortalized human hepatocytes with Tamoxifen-mediated self-recombination. J Hepatol 2007; Jul;47(1):74-82.  PubMed Link

de Piedoue Gm Andrieu-Soler C, Concordet JP, Marisse R, Sun JS, Lopez B, Kuzniak I, Leboulch P, Feugeas JP.  Targeted gene correction with 5' acridine-oligonucleotide conjugates. Oligonucleotides. 2007 Summer;17(2):258-63.  PubMed Link