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1

Efficacité et sécurité du traitement par thérapie génique des patients atteints du syndrome de Wiskott-Aldrich

Galy, Anne, Corre, Guillaume, Cavazzana, Marina, Hacein-Bey-Abina, Salima

Journal:

médecine/sciences

Year:

2015

Language:

french

File:

PDF, 102 KB

french, 2015

2

LMO2-Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1

Hacein-Bey-Abina, S.

Journal:

Year:

2003

Language:

english

File:

PDF, 488 KB

english, 2003

3

A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency

Hacein-Bey-Abina, Salima, Pai, Sung-Yun, Gaspar, H. Bobby, Armant, Myriam, Berry, Charles C., Blanche, Stephane, Bleesing, Jack, Blondeau, Johanna, de Boer, Helen, Buckland, Karen F., Caccavelli, Laur

Journal:

New England Journal of Medicine

Year:

2014

Language:

english

File:

PDF, 585 KB

english, 2014

4

Advances in adoptive immunotherapy to accelerate T-cellular immune reconstitution after HLA-incompatible hematopoietic stem cell transplantation

Reimann, Christian, Dal Cortivo, Liliane, Hacein-Bey-Abina, Salima, Fischer, Alain, André-Schmutz, Isabelle, Cavazzana-Calvo, Marina

Journal:

Year:

2010

Language:

english

File:

PDF, 1.08 MB

english, 2010

5

Découverte d’anticorps anti-SS-A lors du bilan étiologique d’un infarctus cérébral du sujet jeune : quel impact ?

Henriquez, S., Legris, N., Chrétien, P., Hacein-Bey-Abina, S., Henry, J., Denier, C., Noel, N.

Journal:

La Revue de Médecine Interne

Year:

2019

File:

PDF, 119 KB

2019

6

Engineering Next-Generation CAR-T Cells for Better Toxicity Management

Andrea, Alain E., Chiron, Andrada, Bessoles, StÃ©phanie, Hacein-Bey-Abina, Salima

Journal:

International Journal of Molecular Sciences

Year:

2020

File:

PDF, 3.70 MB

2020

7

Gene Therapy of X-Linked Severe Combined Immunodeficiency

Salima Hacein-Bey-Abina, Alain Fischer, Marina Cavazzana-Calvoa

Journal:

International Journal of Hematology

Year:

2002

Language:

english

File:

PDF, 75 KB

english, 2002

8

Gene therapy of metabolic diseases

Alain Fischer, Salima Hacein-Bey-Abina, Marina Cavazzana-Calvo

Journal:

Journal of Inherited Metabolic Disease

Year:

2006

Language:

english

File:

PDF, 188 KB

english, 2006

9

Immune reconstitution after haematopoietic stem cell transplantation: obstacles and anticipated progress

Marina Cavazzana-Calvo, Isabelle André-Schmutz, Liliane Dal Cortivo, Bénédicte Neven, Salima Hacein-Bey-Abina, Alain Fischer

Journal:

Current Opinion in Immunology

Year:

2009

Language:

english

File:

PDF, 126 KB

english, 2009

10

Severe combined immunodeficiency. A model disease for molecular immunology and therapy

Alain Fischer, Françoise Le Deist, Salima Hacein-Bey-Abina, Isabelle André-Schmutz, Geneviève De Saint Basile, Jean-Pierre De Villartay, Marina Cavazzana-Calvo

Journal:

Immunological Reviews

Year:

2005

Language:

english

File:

PDF, 836 KB

english, 2005

11

Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer

Pike-Overzet, K, Rodijk, M, Ng, Y-Y, Baert, M R M, Lagresle-Peyrou, C, Schambach, A, Zhang, F, Hoeben, R C, Hacein-Bey-Abina, S, Lankester, A C

Journal:

Year:

2011

Language:

english

File:

PDF, 944 KB

english, 2011

12

Stem cell self-renewal: lessons from bone marrow, gut and iPS toward clinical applications

Staal, F J T, Baum, C, Cowan, C, Dzierzak, E, Hacein-Bey-Abina, S, Karlsson, S, Lapidot, T, Lemischka, I, Mendez-Ferrer, S, Mikkers, H

Journal:

Year:

2011

Language:

english

File:

PDF, 451 KB

english, 2011

13

20 years of gene therapy for SCID

Fischer, Alain, Hacein-Bey-Abina, Salima, Cavazzana-Calvo, Marina

Journal:

Nature Immunology

Year:

2010

Language:

english

File:

PDF, 2.17 MB

english, 2010

14

Impact of HLA matching on outcome of hematopoietic stem cell transplantation in children with inherited diseases: a single-center comparative analysis of genoidentical, haploidentical or unrelated donors

Caillat-Zucman, S, Le Deist, F, Haddad, E, Gannagé, M, Dal Cortivo, L, Jabado, N, Hacein-Bey-Abina, S, Blanche, S, Casanova, J-L, Fischer, A

Journal:

Bone Marrow Transplantation

Year:

2004

Language:

english

File:

PDF, 153 KB

english, 2004

15

Fragile sites are preferential targets for integrations of MLV vectors in gene therapy

Bester, A C, Schwartz, M, Schmidt, M, Garrigue, A, Hacein-Bey-Abina, S, Cavazzana-Calvo, M, Ben-Porat, N, Von Kalle, C, Fischer, A, Kerem, B

Journal:

Year:

2006

Language:

english

File:

PDF, 92 KB

english, 2006

16

Fragile sites are preferential targets for integrations of MLV vectors in gene therapy

Bester, A C, Schwartz, M, Schmidt, M, Garrigue, A, Hacein-Bey-Abina, S, Cavazzana-Calvo, M, Ben-Porat, N, Kalle, C Von, Fischer, A, Kerem, B

Journal:

Year:

2007

Language:

english

File:

PDF, 38 KB

english, 2007

17

Gene therapy targeting hematopoietic stem cells: From the bench to the bedside

M. Cavazzana-Calvo, S. Hacein-Bey-Abina, A. Garrigue, G. Wang, F. Bushman, A. Fischer

Journal:

Blood Cells, Molecules, and Diseases

Year:

2008

Language:

english

File:

PDF, 59 KB

english, 2008

18

The gene corrected clonal inventory in clinical gene therapy trials

Kerstin Schwarzwaelder, Manfred Schmidt, Annette Deichmann, Steven Howe, Marion G. Ott, Stefan Stein, Ulrich Siler, Salima Hacein-Bey-Abina, Hanno Glimm, Dieter Hoelzer, Marina Cavazzana-Calvo, Adrian

Journal:

Blood Cells, Molecules, and Diseases

Year:

2008

Language:

english

File:

PDF, 53 KB

english, 2008

19

Cytokines and culture medium have a major impact on human in vitro T-cell differentiation

Emmanuelle M. Six, Fatine Benjelloun, Alexandrine Garrigue, Delphine Bonhomme, Estelle Morillon, Julien Rouiller, Laure Cacavelli, Johanna Blondeau, Kheira Beldjord, Salima Hacein-Bey-Abina, Marina Ca

Journal:

Blood Cells, Molecules, and Diseases

Year:

2011

Language:

english

File:

PDF, 1.06 MB

english, 2011

20

Immune reconstitution without graft-versus-host disease after haemopoietic stem-cell transplantation: a phase 1/2 study

Isabelle André-Schmutz, Françoise Le Deist, Salima Hacein-Bey-Abina, Ellen Vitetta, John Schindler, Gaelle Chedeville, Etienne Vilmer, Alain Fischer, Marina Cavazzana-Calvo

Journal:

Year:

2002

Language:

english

File:

PDF, 214 KB

english, 2002

21

Gene therapy for immunodeficiency diseases

Alain Fischer, Salima Hacein-Bey-Abina, Marina Cavazzana-Calvo

Journal:

Seminars in Hematology

Year:

2004

Language:

english

File:

PDF, 130 KB

english, 2004

22

Improving immune reconstitution while preventing graft-versus-host disease in allogeneic stem cell transplantation

Marina Cavazzana-Calvo, Isabelle André-Schmutz, Salima Hacein-Bey-Abina, Danielle Bensoussan, Françoise Le Deist, Alain Fischer

Journal:

Seminars in Hematology

Year:

2002

Language:

english

File:

PDF, 101 KB

english, 2002

23

Sustained Correction of X-Linked Severe Combined Immunodeficiency by ex Vivo Gene Therapy

Hacein-Bey-Abina, Salima, Le Deist, Françoise, Carlier, Frédérique, Bouneaud, Cécile, Hue, Christophe, De Villartay, Jean-Pierre, Thrasher, Adrian J., Wulffraat, Nicolas, Sorensen, Ricardo, Dupuis-Gir

Journal:

Boston Medical and Surgical Journal

Year:

2002

Language:

english

File:

PDF, 211 KB

english, 2002

24

Gene therapy for primary immunodeficiencies: part 1

Marina Cavazzana-Calvo, Alain Fischer, Salima Hacein-Bey-Abina, Alessandro Aiuti

Journal:

Current Opinion in Immunology

Year:

2012

Language:

english

File:

PDF, 239 KB

english, 2012

25

Gene therapy for primary adaptive immune deficiencies

Fischer, Alain, Hacein-Bey-Abina, Salima, Cavazzana-Calvo, Marina

Journal:

Journal of Allergy and Clinical Immunology

Year:

2011

Language:

english

File:

PDF, 90 KB

english, 2011

26

A Serious Adverse Event after Successful Gene Therapy for X-Linked Severe Combined Immunodeficiency

Hacein-Bey-Abina, Salima, von Kalle, Christof, Schmidt, Manfred, Le Deist, Françoise, Wulffraat, Nicolas, McIntyre, Elisabeth, Radford, Isabelle, Villeval, Jean-Luc, Fraser, Christopher C., Cavazzana-

Journal:

Boston Medical and Surgical Journal

Year:

2003

Language:

english

File:

PDF, 152 KB

english, 2003

27

Gene Therapy for Severe Combined Immunodeficiency

Cavazzana-Calvo, Marina, Lagresle, Chantal, Hacein-Bey-Abina, Salima, Fischer, Alain

Journal:

Annual Review of Medicine

Year:

2005

Language:

english

File:

PDF, 327 KB

english, 2005

28

Gene Therapy for Primary Immunodeficiencies

Fischer, Alain, Hacein-Bey-Abina, S., Cavazanna-Calvo, M.

Journal:

Immunology and Allergy Clinics of North America

Year:

2010

Language:

english

File:

PDF, 111 KB

english, 2010

29

Gene therapy of primary T cell immunodeficiencies

Fischer, Alain, Hacein-Bey-Abina, Salima, Cavazzana-Calvo, Marina

Journal:

Year:

2013

Language:

english

File:

PDF, 181 KB

english, 2013

30

Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency

Hacein-Bey-Abina, Salima, Hauer, Julia, Lim, Annick, Picard, Capucine, Wang, Gary P., Berry, Charles C., Martinache, Chantal, Rieux-Laucat, Frédéric, Latour, Sylvain, Belohradsky, Bernd H., Leiva, Lil

Journal:

Boston Medical and Surgical Journal

Year:

2010

Language:

english

File:

PDF, 408 KB

english, 2010

31

Gene Therapy for Primary Immunodeficiencies

Fischer, Alain, Hacein-Bey-Abina, S., Cavazzana-Calvo, M.

Journal:

Hematology/Oncology Clinics of North America

Year:

2011

Language:

english

File:

PDF, 104 KB

english, 2011

32

Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

Cartier, N., Hacein-Bey-Abina, S., Bartholomae, C. C., Veres, G., Schmidt, M., Kutschera, I., Vidaud, M., Abel, U., Dal-Cortivo, L., Caccavelli, L., Mahlaoui, N., Kiermer, V., Mittelstaedt, D., Belles

Journal:

Year:

2009

Language:

english

File:

PDF, 741 KB

english, 2009

33

Gene therapy for inherited immunodeficiency

Touzot, Fabien, Hacein-Bey-Abina, Salima, Fischer, Alain, Cavazzana, Marina

Journal:

Expert Opinion on Biological Therapy

Year:

2014

Language:

english

File:

PDF, 358 KB

english, 2014

34

660. The Effect of the B2 Mutation on Hematopoietic Reconstitution in the French X-SCID Gene Therapy Trial

Gray, John T., Hacein-Bey-Abina, Salima, Cavazzana-Calvo, Marina, Fischer, Alain

Journal:

Molecular Therapy

Year:

2006

Language:

english

File:

PDF, 90 KB

english, 2006

35

Critical Variables affecting clinical-grade production of the self-inactivating gamma-retroviral vector for the treatment of X-linked severe combined immunodeficiency

van der Loo, J C M, Swaney, W P, Grassman, E, Terwilliger, A, Higashimoto, T, Schambach, A, Hacein-Bey-Abina, S, Nordling, D L, Cavazzana-Calvo, M, Thrasher, A J, Williams, D A, Reeves, L, Malik, P

Journal:

Year:

2012

Language:

english

File:

PDF, 285 KB

english, 2012

36

DNA bar coding and pyrosequencing to analyze adverse events in therapeutic gene transfer

Wang, G. P., Garrigue, A., Ciuffi, A., Ronen, K., Leipzig, J., Berry, C., Lagresle-Peyrou, C., Benjelloun, F., Hacein-Bey-Abina, S., Fischer, A., Cavazzana-Calvo, M., Bushman, F. D.

Journal:

Nucleic Acids Research

Year:

2008

Language:

english

File:

PDF, 2.61 MB

english, 2008

37

A method to sequence and quantify DNA integration for monitoring outcome in gene therapy

Brady, T., Roth, S. L., Malani, N., Wang, G. P., Berry, C. C., Leboulch, P., Hacein-Bey-Abina, S., Cavazzana-Calvo, M., Papapetrou, E. P., Sadelain, M., Savilahti, H., Bushman, F. D.

Journal:

Nucleic Acids Research

Year:

2011

Language:

english

File:

PDF, 793 KB

english, 2011

38

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

Hacein-Bey-Abina, Salima, Garrigue, Alexandrine, Wang, Gary P., Soulier, Jean, Lim, Annick, Morillon, Estelle, Clappier, Emmanuelle, Caccavelli, Laure, Delabesse, Eric, Beldjord, Kheira, Asnafi, Vahid

Journal:

Journal of Clinical Investigation

Year:

2008

Language:

english

File:

PDF, 1.38 MB

english, 2008

39

Restoration of Human B-cell Differentiation Into NOD-SCID Mice Engrafted With Gene-corrected CD34+ Cells Isolated From Artemis or RAG1-deficient Patients

Lagresle-Peyrou, Chantal, Benjelloun, Fatine, Hue, Christophe, Andre-Schmutz, Isabelle, Bonhomme, Delphine, Forveille, Monique, Beldjord, Kheira, Hacein-Bey-Abina, Salima, De Villartay, Jean-Pierre, C

Journal:

Molecular Therapy

Year:

2008

Language:

english

File:

PDF, 372 KB

english, 2008

40

Gene therapy for SCID-X1 : Where do we stand?

Cavazzana, Marina, Six, Emmanuelle, Lagresle-Peyrou, Chantal, André-Schmutz, Isabelle, Hacein-Bey-Abina, Salima

Journal:

Human Gene Therapy

Year:

2016

Language:

english

File:

PDF, 514 KB

english, 2016

41

Correction of genetic blood defects by gene transfer

Cavazzana-Calvo, Marina, Hacein-Bey-Abina, Salima

Journal:

Current Opinion in Hematology

Year:

2001

Language:

english

File:

PDF, 96 KB

english, 2001

42

Gene therapy of X-linked severe combined immunodeficiency

Cavazzana-Calvo, Marina, Hacein-Bey-Abina, Salima, Fischer, Alain

Journal:

Current Opinion in Allergy and Clinical Immunology

Year:

2002

Language:

english

File:

PDF, 52 KB

english, 2002

43

425. Production of Gene Corrected T Cell Precursors for Therapy of SCID X1

Lagresle-Peyrou, Chantal, Roudaut, Cécile, Gabrion, Aurélie, Caccavelli, Laure, Sadek, Hanem, Chouteau, Myriam, Reimann, Christian, Hacein-Bey-Abina, Salima, Cavazzana, Marina, André-Schmutz, Isabelle

Journal:

Molecular Therapy

Year:

2016

Language:

english

File:

PDF, 334 KB

english, 2016

44

689. The Content of the More Immature Hematopoietic Stem Cells (HSCs) Is Dependent from the Underlying Genetic Diseases: Consequence on Transduction Efficiencies

Caccavelli, Laure, Roudaut, Cécile, Joigneaux, Maeva, Blondeau, Johanna, Touzot, Fabien, Hacein-Bey-Abina, Salima, Cavazzana, Marina

Journal:

Molecular Therapy

Year:

2016

Language:

english

File:

PDF, 301 KB

english, 2016

45

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, Barto

Journal:

New England Journal of Medicine

Year:

2017

Language:

english

File:

PDF, 593 KB

english, 2017

46

Thérapie génique du déficit immunitaire combiné sévère lié à l’X

Fischer, Alain, Hacein-Bey-Abina, Salima, Cavazzana-Calvo, Marina

Journal:

médecine/sciences

Year:

2004

Language:

french

File:

PDF, 252 KB

french, 2004

47

Assessing the effect of genetic markers on drug immunogenicity from a mechanistic model-based approach

DuhazÃ©, Julianne, Caubet, Miguel, HÃ¤ssler, Signe, Bachelet, Delphine, Allez, Matthieu, Deisenhammer, Florian, Fogdell-Hahn, Anna, Gleizes, Aude, Hacein-Bey-Abina, Salima, Mariette, Xavier, Pallardy,

Journal:

BMC Medical Research Methodology

Year:

2020

Language:

english

File:

PDF, 678 KB

english, 2020

48

A Machine Learning Approach for High-Dimensional Time-to-Event Prediction With Application to Immunogenicity of Biotherapies in the ABIRISK Cohort

DuhazÃ©, Julianne, HÃ¤ssler, Signe, Bachelet, Delphine, Gleizes, Aude, Hacein-Bey-Abina, Salima, Allez, Matthieu, Deisenhammer, Florian, Fogdell-Hahn, Anna, Mariette, Xavier, Pallardy, Marc, BroÃ«t, P

Journal:

Frontiers in Immunology

Year:

2020

File:

PDF, 954 KB

2020

49

Anti-RNP positivity in primary SjÃ¶grenâs syndrome is associated with a more active disease and a more frequent muscular and pulmonary involvement

Abbara, Salam, Seror, Raphaele, Henry, Julien, Chretien, Pascale, Gleizes, Aude, Hacein-Bey-Abina, Salima, Mariette, Xavier, Nocturne, Gaetane

Journal:

Year:

2019

File:

PDF, 270 KB

2019

50

Erythropoietin is a major regulator of thrombopoiesis in thrombopoietin-dependent and -independent contexts

Hacein-Bey-Abina, Salima, Estienne, Machadiya, Bessoles, StÃ©phanie, Echchakir, Hamid, Pederzoli-Ribeil, Magali, Chiron, Andrada, Aldaz-Carroll, Lydia, Leducq, Valentin, Zhang, Yanyan, Souyri, MichÃ¨l

Journal:

Experimental Hematology

Year:

2020

File:

PDF, 1.64 MB

2020