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Publications du STI

​Retrouvez ici la liste des publications des équipes du Service des thérapies innovantes. 

Publié le 29 mai 2019


The integrity of the FOG-2 LXCXE pRb-binding motif is required for small intestine homeostasis.
Goupille O, Kadri Z, Langelé A, Luccantoni S, Badoual C, Leboulch P, Chrétien S.
Enhanced Transduction of Macaca fascicularis Hematopoietic Cells with Chimeric Lentiviral Vectors.
Sii-Felice K, Castillo Padilla J, Relouzat F, Cheuzeville J, Tantawet S, Maouche L, Le Grand R, Leboulch P, Payen E4.
UM171 Enhances Lentiviral Gene Transfer and Recovery of Primitive Human Hematopoietic Cells.
Ngom M, Imren S, Maetzig T, Adair JE, Knapp DJHF, Chagraoui J, Fares I, Bordeleau ME, Sauvageau G, Leboulch P, Eaves C, Humphries RK.
Hemoglobin disorders: lentiviral gene therapy in the starting blocks to enter clinical practice.
Sii-Felice K, Giorgi M, Leboulch P, Payen E.
Gene Therapy in Patients with Transfusion-Dependent Beta-Thalassemia
Thompson AA, Walters MC, Kwiatkowski J, Rasko JEJ, Ribeil JA, Hongeng S, Magrin E, Schiller GJ, Payen E, Semeraro M, Moshous D, Lefrere F, Puy H, Bourget P, Magnani A, Caccavelli L, Diana JS, Suarez F, Monpoux F, Brousse V, Poirot C, Brouzes C, Meritet JF, Pondarré C, Beuzard Y, Chrétien S, Lefebvre T, Teachey DT, Anurathapan U, Ho PJ, von Kalle C, Kletzel M, Vichinsky E, Soni S, Veres G, Negre O, Ross RW, Davidson D, Petrusich A, Sandler L, Asmal M, Hermine O, De Montalembert M, Hacein-Bey-Abina S, Blanche S, Leboulch P, Cavazzana M
 Ex Vivo Selection of Transduced Hematopoietic Stem Cells for Gene Therapy of beta-Hemoglobinopathies
Bhukhai K, de Dreuzy E, Giorgi M, Colomb C, Negre O, Denaro M, Gillet-Legrand B, Cheuzeville J, Paulard A, Trebeden-Negre H, Borwornpinyo S, Sii-Felice K, Maouche L, Down JD, Leboulch P, Payen E.
The LXCXE Retinoblastoma Protein-Binding Motif of FOG-2 Regulates Adipogenesis.
Goupille O., Penglong T., Kadri Z., Granger-Locatelli M., Badoual C. Fucharoen S., Maouche-Chrétien L., Leboulch P., and Chrétien S.
Induced pluripotent stem cell generation from a man carrying a complex chromosomal rearrangement as a genetic model for infertility studies
Mouka A, Izard V, Tachdjian G, Brisset S, Yates F, Mayeur A, Drevillon L, Jarray R, Leboulch P, Maouche-Chretien L, Tosca L
Gene Therapy in a Patient with Sickle Cell Disease
Ribeil JA, Hacein-Bey-Abina S, Payen E, Magnani A, Semeraro M, Magrin E, Caccavelli L, Neven B, Bourget P, El Nemer W, Bartolucci P, Weber L, Puy H, Meritet JF, Grevent D, Beuzard Y, Chretien S, Lefebvre T, Ross RW, Negre O, Veres G, Sandler L, Soni S, de Montalembert M, Blanche S, Leboulch P, Cavazzana M
N. Engl. J. Med., 2017
Gene Therapy in a Patient with Sickle Cell Disease REPLY
Leboulch P, Cavazzana M
N. Engl. J. Med., 2017
Inhibition of the acetyl lysine-binding pocket of bromodomain and extraterminal domain proteins interferes with adipogenesis
Goupille O, Penglong T, Kadri Z, Granger-Locatelli M, Fucharoen S, Maouche-Chretien L, Prost S, Leboulch P, Chretien S.
Gene Therapy of the beta-Hemoglobinopathies by Lentiviral Transfer of the beta(A(T87Q))-Globin Gene
Negre O, Eggimann AV, Beuzard Y, Ribeil JA, Bourget P, Borwornpinyo S, Hongeng S, Hacein-Bey S, Cavazzana M, Leboulch P, Payen E
Hum. Gene Ther., 2016
High-Efficiency Transduction of Primary Human Hematopoietic Stem/Progenitor Cells by AAV6 Vectors: Strategies for Overcoming Donor-Variation and Implications in Genome Editing
Ling C, Bhukhai K, Yin ZF, Tan MQ, Yoder MC, Leboulch P, Payen E, Srivastava A
Sci Rep, 2016
Preclinical Evaluation of Efficacy and Safety of an Improved Lentiviral Vector for the Treatment of beta-Thalassemia and Sickle Cell Disease
Negre O, Bartholomae C, Beuzard Y, Cavazzana M, Christiansen L, Courne C, Deichmann A, Denaro M, De Dreuzy E, Finer M, Fronza R, Gillet-Legrand B, Joubert C, Kutner R, Leboulch P, Maouche L, Paulard A, Pierciey FJ, Rothe M, Ryu B, Schmidt M, von Kalle C, Payen E, Veres G
Curr. Gene Ther., 2015
Erosion of the chronic myeloid leukaemia stem cell pool by PPAR gamma agonists
Prost S, Relouzat F, Spentchian M, Ouzegdouh Y, Saliba J, Massonnet G, Beressi JP, Verhoeyen E, Raggueneau V, Maneglier B, Castaigne S, Chomienne C, Chretien S, Rousselot P, Leboulch P
Nature, 2015
Transplantation of Macaca cynomolgus iPS-derived hematopoietic cells in NSG immunodeficient mice
Abed S, Tubsuwan A, Chaichompoo P, Park IH, Pailleret A, Benyoucef A, Tosca L, De Dreuzy E, Paulard A, Granger-Locatelli M, Relouzat F, Prost S, Tachdjian G, Fucharoen S, Daley GQ, Payen E, Chretien S, Leboulch P, Maouche-Chretien L
Haematologica, 2015
Erythropoietin and IGF-1 signaling synchronize cell proliferation and maturation during erythropoiesis
Kadri Z, Lefevre C, Goupille O, Penglong T, Granger-Locatelli M, Fucharoen S, Maouche-Chretien L, Leboulch P, Chretien S
Genes Dev., 2015
Loss of Major Molecular Response As a Trigger for Restarting Tyrosine Kinase Inhibitor Therapy in Patients With Chronic-Phase Chronic Myelogenous Leukemia Who Have Stopped Imatinib After Durable Undetectable Disease
Rousselot P, Charbonnier A, Cony-Makhoul P, Agape P, Nicolini FE, Varet B, Gardembas M, Etienne G, Rea D, Roy L, Escoffre-Barbe M, Guerci-Bresler A, Tulliez M, Prost S, Spentchian M, Cayuela JM, Reiffers J, Chomel JC, Turhan A, Guilhot J, Guilhot F, Mahon FX
J. Clin. Oncol., 2014
An activin receptor IIA ligand trap corrects ineffective erythropoiesis in beta-thalassemia
Dussiot M, Maciel TT, Fricot A, Chartier C, Negre O, Veiga J, Grapton D, Paubelle E, Payen E, Beuzard Y, Leboulch P, Ribeil JA, Arlet JB, Cote F, Courtois G, Ginzburg YZ, Daniel TO, Chopra R, Sung V, Hermine O, Moura IC
Nat. Med., 2014
HSP70 sequestration by free alpha-globin promotes ineffective erythropoiesis in beta-thalassaemia
Arlet JB, Ribeil JA, Guillem F, Negre O, Hazoume A, Marcion G, Beuzard Y, Dussiot M, Moura IC, Demarest S, de Beauchene IC, Belaid-Choucair Z, Sevin M, Maciel TT, Auclair C, Leboulch P, Chretien S, Tchertanov L, Baudin-Creuza V, Seigneuric R, Fontenay M, Garrido C, Hermine O, Courtois G
Nature, 2014
C-Type Lectin-like Receptor LOX-1 Promotes Dendritic Cell-Mediated Class-Switched B Cell Responses
Joo H, Li DP, Dullaers M, Kim TW, Duluc D, Upchurch K, Xue YM, Zurawski S, Le Grand R, Liu YJ, Kuroda M, Zurawski G, Oh S
Immunity, 2014
Musculoskeletal Pain in Patients With Chronic Myeloid Leukemia After Discontinuation of Imatinib: A Tyrosine Kinase Inhibitor Withdrawal Syndrome? Reply
Rousselot P, Charbonnier A, Cony-Makhoul P, Agape P, Nicolini FE, Varet B, Gardembas M, Etienne G, Rea D, Roy L, Escoffre-Barbe M, Guerci-Bresler A, Tulliez M, Prost S, Spentchian M, Cayuela JM, Reiffers J, Chomel JC, Turhan A, Guilhot J, Guilhot F, Mahon FX
J. Clin. Oncol., 2014
GENE THERAPY Primed for take-off
Leboulch P
Nature, 2013
Parallel Assessment of Globin Lentiviral Transfer in Induced Pluripotent Stem Cells and Adult Hematopoietic Stem Cells Derived from the Same Transplanted beta-Thalassemia Patient
Tubsuwan A, Abed S, Deichmann A, Kardel MD, Bartholoma C, Cheung A, Negre O, Kadri Z, Fucharoen S, von Kalle C, Payen E, Chretien S, Schmidt M, Eaves CJ, Leboulch P, Maouche-Chretien L
Stem Cells, 2013
Arrayed lentiviral barcoding for quantification analysis of hematopoietic dynamics
Grosselin J, Sii-Felice K, Payen E, Chretien S, Roux DT, Leboulch P
Stem Cells, 2013
Payen E, Colomb C, Negre O, Beuzard Y, Hehir K, Leboulch P
Methods Enzymol., 2012
Stem Cell Factor-Displaying Simian Immunodeficiency Viral Vectors Together with a Low Conditioning Regimen Allow for Long-Term Engraftment of Gene-Marked Autologous Hematopoietic Stem Cells in Macaques
Verhoeyen E, Relouzat F, Cambot M, Costa C, Negre D, Legrand F, Joubert C, Le Grand R, Cosset FL, Leboulch P, Dubart-Kupperschmitt A, Prost S
Hum. Gene Ther., 2012
BET bromodomain inhibition rescues erythropoietin differentiation of human erythroleukemia cell line UT7
Goupille O, Penglong T, Lefevre C, Granger M, Kadri Z, Fucharoen S, Maouche-Chretien L, Leboulch P, Chretien S
Biochem. Biophys. Res. Commun., 2012
Advances in stem cell transplantation and gene therapy in the beta-hemoglobinopathies
Payen E, Leboulch P
Hematol.-Am. Soc. Hematol. Educ. Program, 2012