Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease
Esrick, Erica B, Lehmann, Leslie E, Biffi, Alessandra, Achebe, Maureen, Brendel, Christian, Ciuculescu, Marioara F, Daley, Heather, MacKinnon, Brenda, Morris, Emily, Federico, Amy, Abriss, Daniela, Boardman, Kari, Khelladi, Radia, Shaw, Kit, Negre, Helene, Negre, Olivier, Nikiforow, Sarah, Ritz, Jerome, Pai, Sung-Yun, London, Wendy B, Dansereau, Colleen, Heeney, Matthew M, Armant, Myriam, Manis, John P, Williams, David A
Published in The New England journal of medicine (21.01.2021)
Published in The New England journal of medicine (21.01.2021)
Get full text
Journal Article
Gene Therapy in a Patient with Sickle Cell Disease
Ribeil, Jean-Antoine, Hacein-Bey-Abina, Salima, Payen, Emmanuel, Magnani, Alessandra, Semeraro, Michaela, Magrin, Elisa, Caccavelli, Laure, Neven, Benedicte, Bourget, Philippe, El Nemer, Wassim, Bartolucci, Pablo, Weber, Leslie, Puy, Hervé, Meritet, Jean-François, Grevent, David, Beuzard, Yves, Chrétien, Stany, Lefebvre, Thibaud, Ross, Robert W, Negre, Olivier, Veres, Gabor, Sandler, Laura, Soni, Sandeep, de Montalembert, Mariane, Blanche, Stéphane, Leboulch, Philippe, Cavazzana, Marina
Published in The New England journal of medicine (02.03.2017)
Published in The New England journal of medicine (02.03.2017)
Get full text
Journal Article
Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial
Magrin, Elisa, Semeraro, Michaela, Hebert, Nicolas, Joseph, Laure, Magnani, Alessandra, Chalumeau, Anne, Gabrion, Aurélie, Roudaut, Cécile, Marouene, Jouda, Lefrere, Francois, Diana, Jean-Sebastien, Denis, Adeline, Neven, Bénédicte, Funck-Brentano, Isabelle, Negre, Olivier, Renolleau, Sylvain, Brousse, Valentine, Kiger, Laurent, Touzot, Fabien, Poirot, Catherine, Bourget, Philippe, El Nemer, Wassim, Blanche, Stéphane, Tréluyer, Jean-Marc, Asmal, Mohammed, Walls, Courtney, Beuzard, Yves, Schmidt, Manfred, Hacein-Bey-Abina, Salima, Asnafi, Vahid, Guichard, Isabelle, Poirée, Maryline, Monpoux, Fabrice, Touraine, Philippe, Brouzes, Chantal, de Montalembert, Mariane, Payen, Emmanuel, Six, Emmanuelle, Ribeil, Jean-Antoine, Miccio, Annarita, Bartolucci, Pablo, Leboulch, Philippe, Cavazzana, Marina
Published in Nature medicine (01.01.2022)
Published in Nature medicine (01.01.2022)
Get full text
Journal Article
In Vivo Outcome of Homology-Directed Repair at the HBB Gene in HSC Using Alternative Donor Template Delivery Methods
Pattabhi, Sowmya, Lotti, Samantha N., Berger, Mason P., Singh, Swati, Lux, Christopher T., Jacoby, Kyle, Lee, Calvin, Negre, Olivier, Scharenberg, Andrew M., Rawlings, David J.
Published in Molecular therapy. Nucleic acids (06.09.2019)
Published in Molecular therapy. Nucleic acids (06.09.2019)
Get full text
Journal Article
Preclinical evaluation of efficacy and safety of an improved lentiviral vector for the treatment of β-thalassemia and sickle cell disease
Negre, Olivier, Bartholomae, Cynthia, Beuzard, Yves, Cavazzana, Marina, Christiansen, Lauryn, Courne, Céline, Deichmann, Annette, Denaro, Maria, de Dreuzy, Edouard, Finer, Mitchell, Fronza, Raffaele, Gillet-Legrand, Béatrix, Joubert, Christophe, Kutner, Robert, Leboulch, Philippe, Maouche, Leïla, Paulard, Anaïs, Pierciey, Francis J, Rothe, Michael, Ryu, Byoung, Schmidt, Manfred, von Kalle, Christof, Payen, Emmanuel, Veres, Gabor
Published in Current gene therapy (01.01.2015)
Published in Current gene therapy (01.01.2015)
Get more information
Journal Article
TALEN-Mediated Gene Editing of HBG in Human Hematopoietic Stem Cells Leads to Therapeutic Fetal Hemoglobin Induction
Lux, Christopher T, Pattabhi, Sowmya, Berger, Mason, Nourigat, Cynthia, Flowers, David A, Negre, Olivier, Humbert, Olivier, Yang, Julia G, Lee, Calvin, Jacoby, Kyle, Bernstein, Irwin, Kiem, Hans-Peter, Scharenberg, Andrew, Rawlings, David J
Published in Molecular therapy. Methods & clinical development (15.03.2019)
Published in Molecular therapy. Methods & clinical development (15.03.2019)
Get full text
Journal Article
Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy
Brendel, Christian, Negre, Olivier, Rothe, Michael, Guda, Swaroopa, Parsons, Geoff, Harris, Chad, McGuinness, Meaghan, Abriss, Daniela, Tsytsykova, Alla, Klatt, Denise, Bentler, Martin, Pellin, Danilo, Christiansen, Lauryn, Schambach, Axel, Manis, John, Trebeden-Negre, Helene, Bonner, Melissa, Esrick, Erica, Veres, Gabor, Armant, Myriam, Williams, David A.
Published in Molecular therapy. Methods & clinical development (12.06.2020)
Published in Molecular therapy. Methods & clinical development (12.06.2020)
Get full text
Journal Article
P1257: SMART101 DONOR T‐LYMPHOID PROGENITORS TO ACCELERATE IMMUNE RECONSTITUTION POST‐HAPLOIDENTICAL PERIPHERAL BLOOD STEM CELL TRANSPLANTATION WITH POST‐TRANSPLANT CYCLOPHOSPHAMIDE: SI101‐02 PHASE I/II
Ciceri, Fabio, Peffault de Latour, Régis, Devillier, Raynier, Chiusolo, Patrizia, Bauquet, Aurelie, Simons, Laura, Heimendinger, Pierre, Gaudeaux, Pierre, Negre, Olivier, Soheili, Tayebeh‐Shabi, Paillet, Juliette, Andre, Isabelle, Oster, Sebastien, Cavazzana, Marina, Lehmann, Frederic
Published in HemaSphere (08.08.2023)
Published in HemaSphere (08.08.2023)
Get full text
Journal Article
P1403: SI101‐01 PHASE I/II STUDY EVALUATING SAFETY AND EFFICACY OF ALLOGENEIC SMART101 T‐LYMPHOID PROGENITOR INJECTION TO ACCELERATE IMMUNE RECONSTITUTION AFTER T‐CELL DEPLETED ALLOGENEIC HSCT
Jan Boelens, Jaap, Umair Mushtaq, Muhammad, Mcguirk, Joseph, Bauquet, Aurelie, Simons, Laura, Heimendinger, Pierre, Gaudeaux, Pierre, Andre, Isabelle, Paillet, Juliette, Negre, Olivier, Soheili, Tayebeh‐Shabi, Oster, Sebastien, Brink, Marcel R.M., Lehmann, Frederic, Cavazzana, Marina, Perales, Miguel‐Angel
Published in HemaSphere (08.08.2023)
Published in HemaSphere (08.08.2023)
Get full text
Journal Article
Staurosporine Increases Lentiviral Vector Transduction Efficiency of Human Hematopoietic Stem and Progenitor Cells
Lewis, Gretchen, Christiansen, Lauryn, McKenzie, Jessica, Luo, Min, Pasackow, Eli, Smurnyy, Yegor, Harrington, Sean, Gregory, Philip, Veres, Gabor, Negre, Olivier, Bonner, Melissa
Published in Molecular therapy. Methods & clinical development (15.06.2018)
Published in Molecular therapy. Methods & clinical development (15.06.2018)
Get full text
Journal Article
T-Cell Progenitors As A New Immunotherapy to Bypass Hurdles of Allogeneic Hematopoietic Stem Cell Transplantation
Gaudeaux, Pierre, Moirangthem, Ranjita Devi, Bauquet, Aurélie, Simons, Laura, Joshi, Akshay, Cavazzana, Marina, Nègre, Olivier, Soheili, Shabi, André, Isabelle
Published in Frontiers in immunology (07.07.2022)
Published in Frontiers in immunology (07.07.2022)
Get full text
Journal Article
Auto-protective redox buffering systems in stimulated macrophages
Ferret, Pierre-Jacques, Soum, Emmanuelle, Negre, Olivier, Fradelizi, Didier
Published in BMC immunology (12.03.2002)
Published in BMC immunology (12.03.2002)
Get full text
Journal Article
Gene-addition/editing therapy in sickle cell disease
Pollock, Galia, Negre, Olivier, Ribeil, Jean-Antoine
Published in La Presse médicale (1983) (01.12.2023)
Published in La Presse médicale (1983) (01.12.2023)
Get full text
Journal Article
Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β(A(T87Q))-Globin Gene
Negre, Olivier, Eggimann, Anne-Virginie, Beuzard, Yves, Ribeil, Jean-Antoine, Bourget, Philippe, Borwornpinyo, Suparerk, Hongeng, Suradej, Hacein-Bey, Salima, Cavazzana, Marina, Leboulch, Philippe, Payen, Emmanuel
Published in Human gene therapy (01.02.2016)
Published in Human gene therapy (01.02.2016)
Get more information
Journal Article
An activin receptor IIA ligand trap corrects ineffective erythropoiesis in β-thalassemia
Dussiot, Michael, Maciel, Thiago T, Fricot, Aurélie, Chartier, Céline, Negre, Olivier, Veiga, Joel, Grapton, Damien, Paubelle, Etienne, Payen, Emmanuel, Beuzard, Yves, Leboulch, Philippe, Ribeil, Jean-Antoine, Arlet, Jean-Benoit, Coté, Francine, Courtois, Geneviève, Ginzburg, Yelena Z, Daniel, Thomas O, Chopra, Rajesh, Sung, Victoria, Hermine, Olivier, Moura, Ivan C
Published in Nature medicine (01.04.2014)
Published in Nature medicine (01.04.2014)
Get full text
Journal Article
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
CAVAZZANA-CALVO, Marina, PAYEN, Emmanuel, CAVALLESCO, Resy, GILLET-LEGRAND, Beatrix, CACCAVELLI, Laure, SGARRA, Riccardo, MAOUCHE-CHRETIEN, Leila, BERNAUDIN, Francoise, GIROE, Robert, DORAZIO, Ronald, MULDER, Geert-Jan, POLACK, Axel, NEGRE, Olivier, BANK, Arthur, SOULIER, Jean, LARGHERO, Jérôme, KABBARA, Nabil, DALLE, Bruno, GOURMEL, Bernard, SOCIE, Gérard, CHRETIEN, Stany, CARTIER, Nathalie, AUBOURG, Patrick, WANG, Gary, FISCHER, Alain, CORNETTA, Kenneth, GALACTEROS, Frédéric, BEUZARD, Yves, GLUCKMAN, Eliane, BUSHMAN, Frederick, HACEIN-BEY-ABINA, Salima, LEBOULCH, Philippe, HEHIR, Kathleen, FUSIL, Floriane, DOWN, Julian, DENARO, Maria, BRADY, Troy, WESTERMAN, Karen
Published in Nature (London) (16.09.2010)
Published in Nature (London) (16.09.2010)
Get full text
Journal Article
Therapeutically relevant engraftment of a CRISPR-Cas9-edited HSC-enriched population with HbF reactivation in nonhuman primates
Humbert, Olivier, Radtke, Stefan, Samuelson, Clare, Carrillo, Ray R, Perez, Anai M, Reddy, Sowmya S, Lux, Christopher, Pattabhi, Sowmya, Schefter, Lauren E, Negre, Olivier, Lee, Ciaran M, Bao, Gang, Adair, Jennifer E, Peterson, Christopher W, Rawlings, David J, Scharenberg, Andrew M, Kiem, Hans-Peter
Published in Science translational medicine (31.07.2019)
Published in Science translational medicine (31.07.2019)
Get more information
Journal Article
SMART101 Donor T-Lymphoid Progenitors to Accelerate Immune Reconstitution Post-Haploidentical Peripheral Blood Stem Cell Transplantation with Post-Transplant Cyclophosphamide: SI101-02 First-in-Human Phase I/II
Devillier, Raynier, Peffault De Latour, Regis, Chevallier, Patrice, Huynh, Anne, Chiusolo, Patrizia, Bauquet, Aurelie, Simons, Laura, Heimendinger, Pierre, Negre, Olivier, Soheili, Shabi, Paillet, Juliette, Andre, Isabelle, Oster, Sebastien, Cavazzana, Marina, Lehmann, Frédéric François, Ciceri, Fabio
Published in Blood (02.11.2023)
Published in Blood (02.11.2023)
Get full text
Journal Article