Marina Cavazzana

Marina Cavazzana

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Marina Cavazzana

Marina Cavazzana

Publications by authors named "Marina Cavazzana"

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Hematopoietic stem cell transplantation for CD40 ligand deficiency: Results from an EBMT/ESID-IEWP-SCETIDE-PIDTC study.

J Allergy Clin Immunol 2019 Jun 17;143(6):2238-2253. Epub 2019 Jan 17.

Department of Pediatric Immunology and HSCT, Great North Children's Hospital, Newcastle upon Tyne, United Kingdom; Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne, United Kingdom.

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https://linkinghub.elsevier.com/retrieve/pii/S00916749193003
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http://dx.doi.org/10.1016/j.jaci.2018.12.1010DOI Listing
June 2019

Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges.

Nat Rev Drug Discov 2019 Jun;18(6):447-462

INSERM UMR 1163, Laboratory of Human Lymphohematopoiesis, Paris, France.

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http://www.nature.com/articles/s41573-019-0020-9
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http://dx.doi.org/10.1038/s41573-019-0020-9DOI Listing
June 2019

Disease Evolution and Response to Rapamycin in Activated Phosphoinositide 3-Kinase δ Syndrome: The European Society for Immunodeficiencies-Activated Phosphoinositide 3-Kinase δ Syndrome Registry.

Front Immunol 2018 16;9:543. Epub 2018 Mar 16.

Center for Chronic Immunodeficiency, Medical Center - University of Freiburg, Freiburg, Germany.

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http://journal.frontiersin.org/article/10.3389/fimmu.2018.00
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http://dx.doi.org/10.3389/fimmu.2018.00543DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5863269PMC
May 2019

Concise Review: BOOSTING T Cell Reconstitution Following Allogeneic Transplantation-Current Concepts and Future Perspectives.

Stem Cells Transl Med 2019 Mar 18. Epub 2019 Mar 18.

Laboratory of Human Lymphohematopoiesis, INSERM UMR 1163, Imagine Institute, Paris, France.

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http://dx.doi.org/10.1002/sctm.18-0248DOI Listing
March 2019

Baboon envelope LVs efficiently transduced human adult, fetal, and progenitor T cells and corrected SCID-X1 T-cell deficiency.

Blood Adv 2019 Feb;3(3):461-475

Centre International de Recherche en Infectiologie, Université Lyon, Université Claude Bernard Lyon 1, INSERM, U1111, Centre National de la Recherche Scientifique, Unité Mixte de Recherche (UMR) 5308, Ecole Normale Supérieure de Lyon, Lyon, France.

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http://dx.doi.org/10.1182/bloodadvances.2018027508DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6373736PMC
February 2019

Gene Therapy for Hemoglobinopathies.

Hum Gene Ther 2018 10;29(10):1106-1113

1 University of Paris Descartes-Sorbonne Paris Cité , IMAGINE Institute, Paris, France; and University of Modena and Reggio Emilia , Modena, Italy .

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http://dx.doi.org/10.1089/hum.2018.122DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6196755PMC
October 2018

Sickle SCAN™ (BioMedomics) fulfills analytical conditions for neonatal screening of sickle cell disease.

Ann Biol Clin (Paris) 2018 Aug;76(4):416-420

Département de biothérapie, Hôpital universitaire Necker-Enfants malades, AP-HP, Paris, France, Centre d'investigation clinique, Hôpitaux universitaires Paris-Ouest, AP-HP, Inserm, Paris, France.

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http://dx.doi.org/10.1684/abc.2018.1354DOI Listing
August 2018

Generation of adult human T-cell progenitors for immunotherapeutic applications.

J Allergy Clin Immunol 2018 Apr 5;141(4):1491-1494.e4. Epub 2017 Dec 5.

Human Lymphohaematopoiesis Laboratory, INSERM U1163, Paris, France; University of Paris Descartes-Sorbonne Paris Cité, IMAGINE Institute, Paris, France; Biotherapy Clinical Investigation Centre, Necker Children's Hospital, Paris, France. Electronic address:

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http://dx.doi.org/10.1016/j.jaci.2017.10.034DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5887055PMC
April 2018

[Evidence for the widespread use of neonatal screening for sickle cell disease].

Med Sci (Paris) 2018 Apr 16;34(4):309-311. Epub 2018 Apr 16.

Centre d'investigation clinique de biothérapie, hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris, 149, rue de Sèvres, 75015 Paris, France - Service de génétique, hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris, 149, rue de Sèvres, 75015 Paris, France.

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http://dx.doi.org/10.1051/medsci/20183404010DOI Listing
April 2018

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia.

N Engl J Med 2018 04;378(16):1479-1493

From the Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children's Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children's Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia (J.K., D.T.T.); Centenary Institute (J.E.J.R.), University of Sydney, Sydney Medical School (J.E.J.R., P.J.H.), and Royal Prince Alfred Hospital (J.E.J.R., P.J.H.), Camperdown, NSW, Australia; Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (J.-A.R., E.M., D.M., F.L., P.B., A.M., L.C., J.-S.D., F.S., F.M., V.B., C.B., O.H., M.D.M., S.B., M.C.), Groupe Hospitalier Universitaire Ouest (J.-A.R., A.M., L.C., M.C.), IMAGINE Institute (E.M., M.S., D.M., M.C.), Université Paris Descartes (M.S., C. Poirot, S.H.-B.-A.), Université Paris Diderot (H.P., T.L.), Pierre et Marie Curie University (C. Poirot), and Hôpital Cochin (J.-F.M.), Paris, CEA University Paris-Sud, Institute of Emerging Diseases and Innovative Therapies, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Hôpital Louis-Mourier, Colombes (H.P., T.L.), Centre Hospitalier Intercommunal de Créteil, Créteil (C. Pondarré), and Hôpital Bicêtre, Le Kremlin-Bicêtre (S.H.-B.-A.) - all in France; Bluebird Bio, Cambridge (J.-A.R., S.S., G.V., O.N., R.W.R., D.D., A.P., L.S., M.A.), and Harvard Medical School, Brigham and Women's Hospital, Boston (P.L.) - both in Massachusetts; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A., P.L.); and the National Center for Tumor Diseases-German Cancer Research Center, Heidelberg, Germany (C.K.).

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http://dx.doi.org/10.1056/NEJMoa1705342DOI Listing
April 2018

Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study.

J Allergy Clin Immunol 2018 Mar 11;141(3):1036-1049.e5. Epub 2017 Dec 11.

Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, Calif. Electronic address:

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http://dx.doi.org/10.1016/j.jaci.2017.10.041DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6050203PMC
March 2018

Gene Therapy Approaches to Hemoglobinopathies.

Hematol Oncol Clin North Am 2017 10;31(5):835-852

Department of Life Sciences, University of Modena and Reggio Emilia, Via Campi 287, 41125 Modena, Italy. Electronic address:

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http://dx.doi.org/10.1016/j.hoc.2017.06.010DOI Listing
October 2017

Mammalian target of rapamycin inhibition counterbalances the inflammatory status of immune cells in patients with chronic granulomatous disease.

J Allergy Clin Immunol 2017 May 1;139(5):1641-1649.e6. Epub 2016 Oct 1.

Biotherapy Department, Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, AP-HP, Paris, France; University Paris Descartes Sorbonne Paris Cité, Institut Imagine, Paris, France; Inserm UMR 1163, Paris, France. Electronic address:

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http://dx.doi.org/10.1016/j.jaci.2016.08.033DOI Listing
May 2017

Gene Therapy for β-Hemoglobinopathies.

Mol Ther 2017 05 1;25(5):1142-1154. Epub 2017 Apr 1.

Paris Descartes, Sorbonne Paris Cité University, Imagine Institute, 75015 Paris, France; INSERM UMR 1163, Laboratory of Chromatin and Gene Regulation, 75015 Paris, France. Electronic address:

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http://dx.doi.org/10.1016/j.ymthe.2017.03.024DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5417842PMC
May 2017

Gene Therapy in a Patient with Sickle Cell Disease.

N Engl J Med 2017 05;376(21):2094

Imagine Institute, Paris, France

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http://dx.doi.org/10.1056/NEJMc1704009DOI Listing
May 2017

[Gene therapy: many more questions than answers].

Authors:
Marina Cavazzana

Med Sci (Paris) 2017 May 14;33(5):461-462. Epub 2017 Jun 14.

Département de biothérapie, hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris, Paris, France - Centre d'investigation clinique de biothérapie, Groupe hospitalier universitaire Ouest, Assistance Publique-Hôpitaux de Paris, Paris, France - Université Paris Descartes-Sorbonne Paris Cité, Institut Imagine, Paris, France - Inserm UMR 1163, Laboratoire de lymphohématopoïèse humaine, Paris, France.

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http://dx.doi.org/10.1051/medsci/20173305001DOI Listing
May 2017

[Gene therapy for sickle cell disease].

Med Sci (Paris) 2017 May 14;33(5):463-465. Epub 2017 Jun 14.

Département de biothérapie, hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris, Paris, France - Centre d'investigation clinique de biothérapie, Groupe hospitalier universitaire Ouest, Assistance Publique-Hôpitaux de Paris, Inserm, Paris, France - Département d'imunologie pédiatrique-hématologie, Assistance Publique-Hôpitaux de Paris, Paris, France - Université Paris Descartes-Sorbonne Paris Cité, Institut Imagine, Paris, France.

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http://dx.doi.org/10.1051/medsci/20173305002DOI Listing
May 2017

Gene Therapy in a Patient with Sickle Cell Disease.

N Engl J Med 2017 03;376(9):848-855

From the Departments of Biotherapy (J.-A.R., A.M., E.M., L.C., M.C.), Clinical Pharmacy (P. Bourget), Pediatric Neuroradiology (D.G.), General Pediatrics (M.M.), and Pediatric Immunology-Hematology Unit (B.N., S.B.), Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM (J.-A.R., A.M., E.M., L.C., L.W., M.C.), Unité de Technologies Chimiques et Biologiques pour la Santé, Centre National de la Recherche Scientifique Unité Mixte de Recherche 8258, INSERM Unité 1022, Faculté de Pharmacie de Paris, Université Paris Descartes, Chimie ParisTech (S.H.-B.-A.), Immunology Laboratory, Groupe Hospitalier Universitaire Paris-Sud, Hôpital Kremlin-Bicêtre, AP-HP, Le Kremlin-Bicêtre (S.H.-B.-A.), the Institute of Emerging Diseases and Innovative Therapies, Imagine Institute, Université Paris Descartes, Sorbonne Paris Cité University (M.S., B.N., L.W., M.C.), Mère-Enfant Clinical Investigation Center, Groupe Hospitalier Necker Cochin (M.S.), Université Paris Diderot, Sorbonne Paris Cité University, INSERM Institut National de Transfusion Sanguine, Unité Biologie Intégrée du Globule Rouge, Laboratoire d'Excellence GR-Ex (W.E.N.), and Laboratoires de Virologie, Hôpital Cochin (J.-F.M.), Paris, Atomic and Alternative Energy Commission, Université Paris-Sud, Fontenay-aux-Roses (E.P., Y.B., S.C., P.L.), Institut Mondor de Recherche Biomédicale, Equipe 2, Centre de Référence des Syndromes Drépanocytaires Majeurs, Centre Hospitalier Universitaire Henri Mondor, AP-HP, Laboratoire d'Excellence GR-Ex, Créteil (P. Bartolucci), and Université Paris Diderot, Sorbonne Paris Cité University, INSERM Unité 1149, Hôpital Louis-Mourier, AP-HP, Laboratoire d'Excellence GR-Ex, Colombes (H.P., T.L.) - all in France; Bluebird Bio, Cambridge (R.W.R., O.N., G.V., L.S., S.S.), and Brigham and Women's Hospital and Harvard Medical School, Boston (P.L.) - both in Massachusetts; and Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (P.L.).

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http://www.nejm.org/doi/10.1056/NEJMoa1609677
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http://dx.doi.org/10.1056/NEJMoa1609677DOI Listing
March 2017

Limiting Thymic Precursor Supply Increases the Risk of Lymphoid Malignancy in Murine X-Linked Severe Combined Immunodeficiency.

Mol Ther Nucleic Acids 2017 Mar 10;6:1-14. Epub 2016 Dec 10.

Gene Therapy Research Unit, Children's Medical Research Institute, The University of Sydney and The Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia; Discipline of Child and Adolescent Health, The University of Sydney, Westmead, NSW 2145, Australia. Electronic address:

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http://dx.doi.org/10.1016/j.omtn.2016.11.011DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5363493PMC
March 2017

Innovations Needed for Effective Implementation of Gene Therapies.

Authors:
Marina Cavazzana

Front Med (Lausanne) 2017 24;4:29. Epub 2017 Mar 24.

Biotherapy Department, Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; INSERM, Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique-Hôpitaux de Paris, Paris, France; Imagine Institute, Paris Descartes-Sorbonne Paris Cité University, Paris, Italy; INSERM UMR 1163, Laboratory of Human Lymphohematopoiesis, Paris, France.

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http://dx.doi.org/10.3389/fmed.2017.00029DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5364139PMC
March 2017

Gene Therapy with Hematopoietic Stem Cells: The Diseased Bone Marrow's Point of View.

Stem Cells Dev 2017 01 16;26(2):71-76. Epub 2016 Oct 16.

2 Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique-Hôpitaux de Paris, INSERM , Paris, France .

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http://dx.doi.org/10.1089/scd.2016.0230DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5248546PMC
January 2017

X-linked primary immunodeficiency associated with hemizygous mutations in the moesin (MSN) gene.

J Allergy Clin Immunol 2016 12 4;138(6):1681-1689.e8. Epub 2016 Jun 4.

Laboratory of Human Lymphohematopoiesis, INSERM UMR 1163, Paris, France; Paris Descartes-Sorbonne Paris Cité University, Imagine Institute, Paris, France; Biotherapy Clinical Investigation Center, Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France. Electronic address:

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http://dx.doi.org/10.1016/j.jaci.2016.04.032DOI Listing
December 2016

Specific T cells for the treatment of cytomegalovirus and/or adenovirus in the context of hematopoietic stem cell transplantation.

J Allergy Clin Immunol 2016 09 30;138(3):920-924.e3. Epub 2016 Apr 30.

Biotherapy Department, Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; Immunology and Pediatric Hematology Department, Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; Human Lymphohematopoiesis-INSERM UMR1163, Paris Descartes-Sorbonne Paris Cité University, Imagine Institute, Paris, France. Electronic address:

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http://dx.doi.org/10.1016/j.jaci.2016.03.032DOI Listing
September 2016

Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?

Hum Gene Ther 2016 Feb;27(2):108-16

1 Biotherapy Department, Necker Children's Hospital , Assistance Publique-Hôpitaux de Paris, Paris.

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http://dx.doi.org/10.1089/hum.2015.137DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4779287PMC
February 2016

Evaluation of antithymocyte globulin pharmacokinetics and pharmacodynamics in children.

J Allergy Clin Immunol 2016 Jan 17;137(1):306-309.e4. Epub 2015 Jul 17.

Université Paris Descartes-Sorbonne Paris Cité, Institut Imagine, Paris, France; INSERM UMR1163, Paris, France; Département de Biothérapie, Centre d'Investigation Clinique intégré en Biothérapies, Hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris, Paris, France.

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http://search.proquest.com/openview/ca42ac96763a00e67aac143f
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http://linkinghub.elsevier.com/retrieve/pii/S009167491500790
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http://dx.doi.org/10.1016/j.jaci.2015.06.003DOI Listing
January 2016

An in vivo genetic reversion highlights the crucial role of Myb-Like, SWIRM, and MPN domains 1 (MYSM1) in human hematopoiesis and lymphocyte differentiation.

J Allergy Clin Immunol 2015 Dec 26;136(6):1619-1626.e5. Epub 2015 Jul 26.

INSERM UMR 1163, Laboratory of Genome Dynamics in the Immune System, Paris, France; Paris Descartes-Sorbonne Paris Cité University, Imagine Institute, Paris, France. Electronic address:

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http://dx.doi.org/10.1016/j.jaci.2015.06.008DOI Listing
December 2015

[Efficacy and safety of gene therapy for Wiskott-Aldrich syndrome].

Med Sci (Paris) 2015 Dec 16;31(12):1066-9. Epub 2015 Dec 16.

Département de biothérapies, hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris, Paris, France - CIC biothérapies, groupe hospitalier universitaire Ouest, Assistance Publique-Hôpitaux de Paris, Inserm, Paris, France - UTCBS CNRS 8258-Inserm U1022, Faculté des sciences pharmaceutiques et biologiques, université Paris Descartes, Paris, France - Service d'Immunologie Biologique, Groupe Hospitalier Universitaire Paris-Sud, Le-Kremlin-Bicêtre, France.

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http://dx.doi.org/10.1051/medsci/20153112006DOI Listing
December 2015

Severe combined immunodeficiencies and related disorders.

Nat Rev Dis Primers 2015 10 29;1:15061. Epub 2015 Oct 29.

Division of Allergy, Immunology and Blood and Marrow Transplantation, Department of Pediatrics, University of California at San Francisco, San Francisco, California, USA.

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http://dx.doi.org/10.1038/nrdp.2015.61DOI Listing
October 2015

Early-onset hypogammaglobulinemia: A survey of 44 patients.

J Allergy Clin Immunol 2015 Oct 8;136(4):1097-9.e2. Epub 2015 May 8.

INSERM UMR 1163, Paris, France; Paris Descartes University - Sorbonne Paris Cité, Imagine Institute, Paris, France. Electronic address:

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http://dx.doi.org/10.1016/j.jaci.2015.03.038DOI Listing
October 2015

Gene therapy for Wiskott-Aldrich Syndrome.

Curr Gene Ther 2014 ;14(6):413-21

TIGET, Via Olgettina 58, 20132 Milan, Italy.

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July 2015

Comparing genotoxic signatures in cord blood cells from neonates exposed in utero to zidovudine or tenofovir.

AIDS 2015 Jul;29(11):1319-24

aInstitut National de la Santé et de la Recherche Médicale (INSERM), U1163, Université Paris Descartes, Sorbonne Paris Cité, Institut Imagine bLaboratoire d'Hématologie Biologique, Assistance Publique-Hopitaux de Paris (AP-HP) cLaboratoire Génome et Cancer, INSERM, U944 and UMR7212, Hôpital Saint Louis dService de Gynécologie Obstétrique, Hôpital Louis Mourier, Hôpitaux Universitaire Paris Nord Val de Seine (HUPNVS), AP-HP, Colombes eINSERM U1018, Centre de recherche en Epidémiologie et Santé des Populations fUniversité Paris-Sud, Le Kremlin Bicêtre gService de Gynécologie Obstétrique, Hôpital Antoine Béclère, AP-HP, Clamart hService de Gynécologie Obstétrique, Hôpital Bichat, HUPNVS, AP-HP iUniversité Paris-Diderot jInstitut Universitaire d'Hématologie, Paris kDépartement de Biothérapie lUnité d'Immunologie Hématologie Rhumatologie Pédiatrique, Hôpital Necker Enfants Malades, AP-HP mEA 7323, Pharmacologie et évaluation des médicaments chez l'enfant et la femme enceinte, Université Paris Descartes, Sorbonne Paris Cité, Paris, France. *Stéphane Blanche and Isabelle André-Schmutz contributed equally to the writing of this article.

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http://dx.doi.org/10.1097/QAD.0000000000000564DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4502990PMC
July 2015

Hepatitis E virus in hematopoietic stem cell donors: Towards a systematic HEV screening of donors?

J Infect 2015 Jul 26;71(1):141-4. Epub 2015 Feb 26.

Unité d'Immunologie, Hématologie et Rhumatologie pédiatriques, AP-HP, Hôpital Necker - Enfants malades, Paris, France; Institut Imagine, Université Paris Descartes, Sorbonne Paris Cité, Paris, France; EA08, Université Paris Descartes, Sorbonne Paris Cité, Paris, France.

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http://dx.doi.org/10.1016/j.jinf.2015.02.008DOI Listing
July 2015

Systematic neonatal screening for severe combined immunodeficiency and severe T-cell lymphopenia: Analysis of cost-effectiveness based on French real field data.

J Allergy Clin Immunol 2015 Jun 1;135(6):1589-93. Epub 2015 Apr 1.

URC Eco (Clinical Research Unit in Health Economics), Assistance Publique-Hôpitaux de Paris, Hôtel Dieu Hospital, Paris, France; Public Health Unit, Henri Mondor Hospital, Assistance Publique-Hôpitaux de Paris, INSERM UMR 1123, Créteil, France. Electronic address:

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http://dx.doi.org/10.1016/j.jaci.2015.02.004DOI Listing
June 2015

Faster T-cell development following gene therapy compared with haploidentical HSCT in the treatment of SCID-X1.

Blood 2015 Jun 13;125(23):3563-9. Epub 2015 Apr 13.

Département de Biothérapie, Centre d'Investigation Clinique intégré en Biothérapies, Hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris, Paris, France; Université Paris Descartes-Sorbonne Paris Cité, Institut Imagine, Paris, France; INSERM UMR1163, Paris, France;

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http://dx.doi.org/10.1182/blood-2014-12-616003DOI Listing
June 2015

CD45RA depletion in HLA-mismatched allogeneic hematopoietic stem cell transplantation for primary combined immunodeficiency: A preliminary study.

J Allergy Clin Immunol 2015 May 3;135(5):1303-9.e1-3. Epub 2014 Oct 3.

Département de Biothérapie, Centre d'Investigation Clinique intégré en Biothérapies, Hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France; Université Paris Descartes-Sorbonne Paris Cité, Institut Imagine, Paris, France; INSERM UMR1163, Paris, France.

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http://dx.doi.org/10.1016/j.jaci.2014.08.019DOI Listing
May 2015

An inherited immunoglobulin class-switch recombination deficiency associated with a defect in the INO80 chromatin remodeling complex.

J Allergy Clin Immunol 2015 Apr 11;135(4):998-1007.e6. Epub 2014 Oct 11.

INSERM UMR 1163, The Human Lymphohematopoiesis Laboratory, Imagine Institute, Paris, France; Paris Descartes Sorbonne Paris Cité University, Imagine Institute, Paris, France; Department of Immunology and Hematology, Hôpital Necker Enfants Malades, F-75015 Paris, Paris, France. Electronic address:

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http://dx.doi.org/10.1016/j.jaci.2014.08.030DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4382329PMC
April 2015

Stem cell transplantation for primary immunodeficiencies: the European experience.

Curr Opin Allergy Clin Immunol 2014 Dec;14(6):516-20

aBiotherapy Department, Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris bBiotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique-Hôpitaux de Paris, INSERM cParis Descartes - Sorbonne Paris Cité University, Imagine Institute dINSERM UMR 1163, Laboratory of Human Lymphohematopoiesis eImmunology and Pediatric Hematology Department, Assistance Publique-Hôpitaux de Paris fCollège de France, Paris, France.

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http://dx.doi.org/10.1097/ACI.0000000000000119DOI Listing
December 2014

Circulating endothelial cells as markers of endothelial dysfunction during hematopoietic stem cell transplantation for pediatric primary immunodeficiency.

J Allergy Clin Immunol 2014 Nov 16;134(5):1203-6. Epub 2014 Jul 16.

Service d'Hématologie Biologique, Hôpital George Pompidou, Assistance Publique-Hôpitaux de Paris, Paris, France; Université Paris Descartes-Sorbonne Paris Cité, Paris, France.

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https://linkinghub.elsevier.com/retrieve/pii/S00916749140080
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http://dx.doi.org/10.1016/j.jaci.2014.05.039DOI Listing
November 2014

A modified γ-retrovirus vector for X-linked severe combined immunodeficiency.

N Engl J Med 2014 Oct;371(15):1407-17

From the Departments of Biotherapy (S.H.-B.-A., J. Blondeau, L.C., F.T., M.C.) and Immunology and Pediatric Hematology (S.B., G.C., D.M., B.N., C.P., F.T., A.F.) and the Centre d'Étude des Déficits Immunitaires (C.P.), Hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (AP-HP), the Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM (S.H.-B.-A., J. Blondeau, L.C., F.T., M.C.), Unité de Technologies Chimiques et Biologiques pour la Santé, Centre National de la Recherche Scientifique, 8258-INSERM Unité 1022, Faculté des Sciences Pharmaceutiques et Biologiques, Université Paris Descartes (S.H.-B.-A.), Immunology Laboratory, Groupe Hospitalier Universitaire Paris-Sud, AP-HP, Le Kremlin-Bicêtre (S.H.-B.-A.), Imagine Institute, Paris Descartes-Sorbonne Paris Cité University (S.B., J. Blondeau, L.C., D.M., B.N., C.P., E.S., A.F., M.C.), INSERM Unités Mixtes de Recherche 1163, Laboratory of Human Lymphohematopoiesis (J. Blondeau, L.C., E.S., F.T., A.F., M.C.), Groupe Immunoscope, Immunology Department, Institut Pasteur (A.L.), and Collège de France (A.F.) - all in Paris; Division of Hematology-Oncology (S.-Y.P., H.B., D.G., C.E.H., G.H., L.E.L., W.B.L., D.A.W.) and Division of Immunology (L.D.N.), Boston Children's Hospital, Department of Pediatric Oncology, Dana-Farber Cancer Institute (S.-Y.P., D.G., L.E.L., W.B.L., D.A.W.), Harvard Medical School (S.-Y.P., M.A., L.E.L., W.B.L., J.R., L.E.S., A.T., L.D.N., D.A.W.), Center for Human Cell Therapy, Program in Cellular and Molecular Medicine, Boston Children's Hospital (M.A., J.R., L.E.S., A.T.), Division of Hematologic Malignancies, Dana-Farber Cancer Institute (J.R.), and the Manton Center for Orphan Disease Research (L.D.N.) - all in Boston; Great Ormond Street Hospital for Children NHS Foundation Trust (H.B.G., J.X.-B., A.J.T.) and Section of Molecular and Cellular Immunology, University College London Institute of Child Health (H.B.G., K.F.B., A.

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http://dx.doi.org/10.1056/NEJMoa1404588DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4274995PMC
October 2014

The BLNK adaptor protein has a nonredundant role in human B-cell differentiation.

J Allergy Clin Immunol 2014 Jul 28;134(1):145-54. Epub 2014 Feb 28.

Centre d'Immunologie de Marseille-Luminy, Faculté des Sciences de Luminy, Aix Marseille Université, UM2, Marseille, France; INSERM, U1104, Marseille, France; Centre National de la Recherche Scientifique, UMR7280, Marseille, France. Electronic address:

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https://linkinghub.elsevier.com/retrieve/pii/S00916749140007
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http://dx.doi.org/10.1016/j.jaci.2013.12.1083DOI Listing
July 2014

Successful RAG1-SCID gene therapy depends on the level of RAG1 expression.

J Allergy Clin Immunol 2014 Jul 26;134(1):242-3. Epub 2014 Jun 26.

Department of Immunohematology and Blood Transfusion, Leiden University Medical Center, Leiden, The Netherlands. Electronic address:

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http://dx.doi.org/10.1016/j.jaci.2014.04.033DOI Listing
July 2014

Gene therapy for inherited immunodeficiency.

Expert Opin Biol Ther 2014 Jun 8;14(6):789-98. Epub 2014 Mar 8.

U768 INSERM , Paris , France.

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http://dx.doi.org/10.1517/14712598.2014.895811DOI Listing
June 2014

Hematopoietic stem cell gene therapy: progress on the clinical front.

Authors:
Marina Cavazzana

Hum Gene Ther 2014 Mar 28;25(3):165-70. Epub 2014 Feb 28.

INSERM U1163, 75654 Paris, France .

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http://dx.doi.org/10.1089/hum.2014.2504DOI Listing
March 2014