Format
Sort by
Items per page

Send to

Choose Destination

Search results

Items: 1 to 50 of 106

1.

Autologous Stem-Cell-Based Gene Therapy for Inherited Disorders: State of the Art and Perspectives.

Staal FJT, Aiuti A, Cavazzana M.

Front Pediatr. 2019 Oct 31;7:443. doi: 10.3389/fped.2019.00443. eCollection 2019. Review.

2.

Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID.

Charrier S, Lagresle-Peyrou C, Poletti V, Rothe M, Cédrone G, Gjata B, Mavilio F, Fischer A, Schambach A, de Villartay JP, Cavazzana M, Hacein-Bey-Abina S, Galy A.

Mol Ther Methods Clin Dev. 2019 Sep 13;15:232-245. doi: 10.1016/j.omtm.2019.08.014. eCollection 2019 Dec 13.

3.

Extensive multilineage analysis in patients with mixed chimerism after allogeneic transplantation for sickle cell disease: insight into hematopoiesis and engraftment thresholds for gene therapy.

Magnani A, Pondarré C, Bouazza N, Magalon J, Miccio A, Six E, Roudaut C, Arnaud C, Kamdem A, Touzot F, Gabrion A, Magrin E, Couzin C, Fusaro M, André I, Vernant JP, Gluckman E, Bernaudin F, Bories D, Cavazzana M.

Haematologica. 2019 Sep 19. pii: haematol.2019.227561. doi: 10.3324/haematol.2019.227561. [Epub ahead of print]

4.

Lentiviral and genome-editing strategies for the treatment of β-hemoglobinopathies.

Magrin E, Miccio A, Cavazzana M.

Blood. 2019 Oct 10;134(15):1203-1213. doi: 10.1182/blood.2019000949.

PMID:
31467062
5.

Ex vivo generated human T-lymphoid progenitors as a tool to accelerate immune reconstitution after partially HLA compatible hematopoietic stem cell transplantation or after gene therapy.

André I, Simons L, Ma K, Moirangthem RD, Diana JS, Magrin E, Couzin C, Magnani A, Cavazzana M.

Bone Marrow Transplant. 2019 Aug;54(Suppl 2):749-755. doi: 10.1038/s41409-019-0599-9. Review.

PMID:
31431705
6.

Gene therapy of hemoglobinopathies: progress and future challenges.

Ikawa Y, Miccio A, Magrin E, Kwiatkowski JL, Rivella S, Cavazzana M.

Hum Mol Genet. 2019 Oct 1;28(R1):R24-R30. doi: 10.1093/hmg/ddz172.

PMID:
31322165
7.

Risk factors and outcomes according to age at transplantation with an HLA-identical sibling for sickle cell disease.

Cappelli B, Volt F, Tozatto-Maio K, Scigliuolo GM, Ferster A, Dupont S, Simões BP, Al-Seraihy A, Aljurf MD, Almohareb F, Belendez C, Matthes S, Dhedin N, Pondarre C, Dalle JH, Bertrand Y, Vannier JP, Kuentz M, Lutz P, Michel G, Rafii H, Neven B, Zecca M, Bader P, Cavazzana M, Labopin M, Locatelli F, Magnani A, Ruggeri A, Rocha V, Bernaudin F, de La Fuente J, Corbacioglu S, Gluckman E; Eurocord, the Cellular Therapy and Immunobiology Working Party (CTIWP) and the Paediatric Diseases Working Party (PDWP) of the EBMT.

Haematologica. 2019 Dec;104(12):e543-e546. doi: 10.3324/haematol.2019.216788. Epub 2019 Apr 24. No abstract available.

8.

Safety of CD34+ Hematopoietic Stem Cells and CD4+ T Lymphocytes Transduced with LVsh5/C46 in HIV-1 Infected Patients with High-Risk Lymphoma.

Delville M, Touzot F, Couzin C, Hmitou I, Djerroudi L, Ouedrani A, Lefrère F, Tuchman-Durand C, Mollet C, Fabreguettes JR, Ferry N, Laganier L, Magnani A, Magrin E, Jolaine V, Saez-Cirion A, Wolstein O, Symonds G, Frange P, Moins-Teisserenc H, Chaix-Baudier ML, Toubert A, Larghero J, Parquet N, Brignier AC, Barré-Sinoussi F, Oksenhendler E, Cavazzana M.

Mol Ther Methods Clin Dev. 2019 Feb 26;13:303-309. doi: 10.1016/j.omtm.2019.02.006. eCollection 2019 Jun 14.

9.

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

Simons L, Cavazzana M, André I.

Stem Cells Transl Med. 2019 Jul;8(7):650-657. doi: 10.1002/sctm.18-0248. Epub 2019 Mar 18. Review.

10.

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

Cavazzana M, Bushman FD, Miccio A, André-Schmutz I, Six E.

Nat Rev Drug Discov. 2019 Jun;18(6):447-462. doi: 10.1038/s41573-019-0020-9. Review.

PMID:
30858502
11.

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

Bernadin O, Amirache F, Girard-Gagnepain A, Moirangthem RD, Lévy C, Ma K, Costa C, Nègre D, Reimann C, Fenard D, Cieslak A, Asnafi V, Sadek H, Mhaidly R, Cavazzana M, Lagresle-Peyrou C, Cosset FL, André I, Verhoeyen E.

Blood Adv. 2019 Feb 12;3(3):461-475. doi: 10.1182/bloodadvances.2018027508.

12.

Innovative Curative Treatment of Beta Thalassemia: Cost-Efficacy Analysis of Gene Therapy Versus Allogenic Hematopoietic Stem-Cell Transplantation.

Coquerelle S, Ghardallou M, Rais S, Taupin P, Touzot F, Boquet L, Blanche S, Benaouadi S, Brice T, Tuchmann-Durand C, Ribeil JA, Magrin E, Lissillour E, Rochaix L, Cavazzana M, Durand-Zaleski I.

Hum Gene Ther. 2019 Jun;30(6):753-761. doi: 10.1089/hum.2018.178. Epub 2019 May 3.

PMID:
30700149
13.

Successful in utero stem cell transplantation in X-linked severe combined immunodeficiency.

Magnani A, Jouannic JM, Rosain J, Gabrion A, Touzot F, Roudaut C, Kracker S, Mahlaoui N, Toubert A, Clave E, Macintyre EA, Radford-Weiss I, Alcantara M, Magrin E, Ternaux B, Nisoy J, Caccavelli L, Darras AM, Picard C, Blanche S, Cavazzana M.

Blood Adv. 2019 Feb 12;3(3):237-241. doi: 10.1182/bloodadvances.2018023176. No abstract available.

14.

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

Ferrua F, Galimberti S, Courteille V, Slatter MA, Booth C, Moshous D, Neven B, Blanche S, Cavazzana M, Laberko A, Shcherbina A, Balashov D, Soncini E, Porta F, Al-Mousa H, Al-Saud B, Al-Dhekri H, Arnaout R, Formankova R, Bertrand Y, Lange A, Smart J, Wolska-Kusnierz B, Aquino VM, Dvorak CC, Fasth A, Fouyssac F, Heilmann C, Hoenig M, Schuetz C, Kelečić J, Bredius RGM, Lankester AC, Lindemans CA, Suarez F, Sullivan KE, Albert MH, Kałwak K, Barlogis V, Bhatia M, Bordon V, Czogala W, Alonso L, Dogu F, Gozdzik J, Ikinciogullari A, Kriván G, Ljungman P, Meyts I, Mustillo P, Smith AR, Speckmann C, Sundin M, Keogh SJ, Shaw PJ, Boelens JJ, Schulz AS, Sedlacek P, Veys P, Mahlaoui N, Janda A, Davies EG, Fischer A, Cowan MJ, Gennery AR; SCETIDE, PIDTC, EBMT & ESID IEWP.

J Allergy Clin Immunol. 2019 Jun;143(6):2238-2253. doi: 10.1016/j.jaci.2018.12.1010. Epub 2019 Jan 17.

15.

Loss of ARHGEF1 causes a human primary antibody deficiency.

Bouafia A, Lofek S, Bruneau J, Chentout L, Lamrini H, Trinquand A, Deau MC, Heurtier L, Meignin V, Picard C, Macintyre E, Alibeu O, Bras M, Molina TJ, Cavazzana M, André-Schmutz I, Durandy A, Fischer A, Oksenhendler E, Kracker S.

J Clin Invest. 2019 Mar 1;129(3):1047-1060. doi: 10.1172/JCI120572. Epub 2019 Feb 4.

16.

T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency.

Clarke EL, Connell AJ, Six E, Kadry NA, Abbas AA, Hwang Y, Everett JK, Hofstaedter CE, Marsh R, Armant M, Kelsen J, Notarangelo LD, Collman RG, Hacein-Bey-Abina S, Kohn DB, Cavazzana M, Fischer A, Williams DA, Pai SY, Bushman FD.

Genome Med. 2018 Sep 28;10(1):70. doi: 10.1186/s13073-018-0580-z.

17.

Gene Therapy for Hemoglobinopathies.

Cavazzana M, Mavilio F.

Hum Gene Ther. 2018 Oct;29(10):1106-1113. doi: 10.1089/hum.2018.122. Review.

18.

A Nontoxic Transduction Enhancer Enables Highly Efficient Lentiviral Transduction of Primary Murine T Cells and Hematopoietic Stem Cells.

Delville M, Soheili T, Bellier F, Durand A, Denis A, Lagresle-Peyrou C, Cavazzana M, Andre-Schmutz I, Six E.

Mol Ther Methods Clin Dev. 2018 Aug 8;10:341-347. doi: 10.1016/j.omtm.2018.08.002. eCollection 2018 Sep 21.

19.

An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype.

Weber L, Poletti V, Magrin E, Antoniani C, Martin S, Bayard C, Sadek H, Felix T, Meneghini V, Antoniou MN, El-Nemer W, Mavilio F, Cavazzana M, Andre-Schmutz I, Miccio A.

Mol Ther Methods Clin Dev. 2018 Aug 4;10:268-280. doi: 10.1016/j.omtm.2018.07.012. eCollection 2018 Sep 21.

20.

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

Nguyen-Khoa T, Mine L, Allaf B, Ribeil JA, Remus C, Stanislas A, Gauthereau V, Enouz S, Kim JS, Yang X, Gluckman E, Beaudeux JL, Munnich A, Girot R, Cavazzana M.

Ann Biol Clin (Paris). 2018 Aug 1;76(4):416-420. doi: 10.1684/abc.2018.1354.

PMID:
29976532
21.

Gene Therapy in Patients with Transfusion-Dependent β-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.

N Engl J Med. 2018 Apr 19;378(16):1479-1493. doi: 10.1056/NEJMoa1705342.

22.

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

Cavazzana M, Stanislas A, Rémus C, Duwez P, Renoult J, Cretet J, Fernandes S, Le Mée C, Allaf B, Porquet D, Munnich A, Polak M, Gauthereau V, Girot R.

Med Sci (Paris). 2018 Apr;34(4):309-311. doi: 10.1051/medsci/20183404010. Epub 2018 Apr 16. French. No abstract available.

23.

Transfusion-related adverse events are decreased in pregnant women with sickle cell disease by a change in policy from systematic transfusion to prophylactic oxygen therapy at home: A retrospective survey by the international sickle cell disease observatory.

Ribeil JA, Labopin M, Stanislas A, Deloison B, Lemercier D, Habibi A, Albinni S, Charlier C, Lortholary O, Lefrere F, De Montalembert M, Blanche S, Galactéros F, Tréluyer JM, Gluckman E, Ville Y, Joseph L, Delville M, Benachi A, Cavazzana M.

Am J Hematol. 2018 Jun;93(6):794-802. doi: 10.1002/ajh.25097. Epub 2018 Apr 17.

24.

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.

Maccari ME, Abolhassani H, Aghamohammadi A, Aiuti A, Aleinikova O, Bangs C, Baris S, Barzaghi F, Baxendale H, Buckland M, Burns SO, Cancrini C, Cant A, Cathébras P, Cavazzana M, Chandra A, Conti F, Coulter T, Devlin LA, Edgar JDM, Faust S, Fischer A, Garcia-Prat M, Hammarström L, Heeg M, Jolles S, Karakoc-Aydiner E, Kindle G, Kiykim A, Kumararatne D, Grimbacher B, Longhurst H, Mahlaoui N, Milota T, Moreira F, Moshous D, Mukhina A, Neth O, Neven B, Nieters A, Olbrich P, Ozen A, Pachlopnik Schmid J, Picard C, Prader S, Rae W, Reichenbach J, Rusch S, Savic S, Scarselli A, Scheible R, Sediva A, Sharapova SO, Shcherbina A, Slatter M, Soler-Palacin P, Stanislas A, Suarez F, Tucci F, Uhlmann A, van Montfrans J, Warnatz K, Williams AP, Wood P, Kracker S, Condliffe AM, Ehl S.

Front Immunol. 2018 Mar 16;9:543. doi: 10.3389/fimmu.2018.00543. eCollection 2018.

25.

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus.

Antoniani C, Meneghini V, Lattanzi A, Felix T, Romano O, Magrin E, Weber L, Pavani G, El Hoss S, Kurita R, Nakamura Y, Cradick TJ, Lundberg AS, Porteus M, Amendola M, El Nemer W, Cavazzana M, Mavilio F, Miccio A.

Blood. 2018 Apr 26;131(17):1960-1973. doi: 10.1182/blood-2017-10-811505. Epub 2018 Mar 8.

PMID:
29519807
26.

Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion.

Lagresle-Peyrou C, Lefrère F, Magrin E, Ribeil JA, Romano O, Weber L, Magnani A, Sadek H, Plantier C, Gabrion A, Ternaux B, Félix T, Couzin C, Stanislas A, Tréluyer JM, Lamhaut L, Joseph L, Delville M, Miccio A, André-Schmutz I, Cavazzana M.

Haematologica. 2018 May;103(5):778-786. doi: 10.3324/haematol.2017.184788. Epub 2018 Feb 22.

27.

Gene transfer into hematopoietic stem cells reduces HLH manifestations in a murine model of Munc13-4 deficiency.

Soheili T, Durand A, Sepulveda FE, Rivière J, Lagresle-Peyrou C, Sadek H, de Saint Basile G, Martin S, Mavilio F, Cavazzana M, André-Schmutz I.

Blood Adv. 2017 Dec 21;1(27):2781-2789. doi: 10.1182/bloodadvances.2017012088. eCollection 2017 Dec 26.

28.

Successful haematopoietic stem cell transplantation in a case of pulmonary alveolar proteinosis due to GM-CSF receptor deficiency.

Frémond ML, Hadchouel A, Schweitzer C, Berteloot L, Bruneau J, Bonnet C, Cros G, Briand C, Magnani A, Pochon C, Delacourt C, Cavazzana M, Moshous D, Fischer A, Blanche S, Blic J, Neven B.

Thorax. 2018 Jun;73(6):590-592. doi: 10.1136/thoraxjnl-2017-211076. Epub 2017 Dec 20. No abstract available.

PMID:
29263235
29.

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

Barzaghi F, Amaya Hernandez LC, Neven B, Ricci S, Kucuk ZY, Bleesing JJ, Nademi Z, Slatter MA, Ulloa ER, Shcherbina A, Roppelt A, Worth A, Silva J, Aiuti A, Murguia-Favela L, Speckmann C, Carneiro-Sampaio M, Fernandes JF, Baris S, Ozen A, Karakoc-Aydiner E, Kiykim A, Schulz A, Steinmann S, Notarangelo LD, Gambineri E, Lionetti P, Shearer WT, Forbes LR, Martinez C, Moshous D, Blanche S, Fisher A, Ruemmele FM, Tissandier C, Ouachee-Chardin M, Rieux-Laucat F, Cavazzana M, Qasim W, Lucarelli B, Albert MH, Kobayashi I, Alonso L, Diaz De Heredia C, Kanegane H, Lawitschka A, Seo JJ, Gonzalez-Vicent M, Diaz MA, Goyal RK, Sauer MG, Yesilipek A, Kim M, Yilmaz-Demirdag Y, Bhatia M, Khlevner J, Richmond Padilla EJ, Martino S, Montin D, Neth O, Molinos-Quintana A, Valverde-Fernandez J, Broides A, Pinsk V, Ballauf A, Haerynck F, Bordon V, Dhooge C, Garcia-Lloret ML, Bredius RG, Kałwak K, Haddad E, Seidel MG, Duckers G, Pai SY, Dvorak CC, Ehl S, Locatelli F, Goldman F, Gennery AR, Cowan MJ, Roncarolo MG, Bacchetta R; Primary Immune Deficiency Treatment Consortium (PIDTC) and the Inborn Errors Working Party (IEWP) of the European Society for Blood and Marrow Transplantation (EBMT).

J Allergy Clin Immunol. 2018 Mar;141(3):1036-1049.e5. doi: 10.1016/j.jaci.2017.10.041. Epub 2017 Dec 11.

30.

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

Simons L, Ma K, de Chappedelaine C, Moiranghtem RD, Elkaim E, Olivré J, Susini S, Appourchaux K, Reimann C, Sadek H, Pellé O, Cagnard N, Magrin E, Lagresle-Peyrou C, Taghon T, Rausell A, Cavazzana M, André-Schmutz I.

J Allergy Clin Immunol. 2018 Apr;141(4):1491-1494.e4. doi: 10.1016/j.jaci.2017.10.034. Epub 2017 Dec 5. No abstract available.

31.

Activation-induced cytidine deaminase prevents pro-B cell acute lymphoblastic leukemia by functioning as a negative regulator in Rag1 deficient pro-B cells.

Auer F, Ingenhag D, Pinkert S, Kracker S, Hacein-Bey-Abina S, Cavazzana M, Gombert M, Martin-Lorenzo A, Lin MH, Vicente-Dueñas C, Sánchez-García I, Borkhardt A, Hauer J.

Oncotarget. 2017 Sep 7;8(44):75797-75807. doi: 10.18632/oncotarget.20563. eCollection 2017 Sep 29.

32.

Gene Therapy Approaches to Hemoglobinopathies.

Ferrari G, Cavazzana M, Mavilio F.

Hematol Oncol Clin North Am. 2017 Oct;31(5):835-852. doi: 10.1016/j.hoc.2017.06.010. Review.

PMID:
28895851
33.

[Gene therapy for sickle cell disease].

Ribeil JA, Blanche S, Cavazzana M.

Med Sci (Paris). 2017 May;33(5):463-465. doi: 10.1051/medsci/20173305002. Epub 2017 Jun 14. French. No abstract available.

34.

[Gene therapy: many more questions than answers].

Cavazzana M.

Med Sci (Paris). 2017 May;33(5):461-462. doi: 10.1051/medsci/20173305001. Epub 2017 Jun 14. French. No abstract available.

35.

Gene Therapy in a Patient with Sickle Cell Disease.

Leboulch P, Cavazzana M.

N Engl J Med. 2017 May 25;376(21):2094. doi: 10.1056/NEJMc1704009. No abstract available.

PMID:
28538131
36.

Mutations in the adaptor-binding domain and associated linker region of p110δ cause Activated PI3K-δ Syndrome 1 (APDS1).

Heurtier L, Lamrini H, Chentout L, Deau MC, Bouafia A, Rosain J, Plaza JM, Parisot M, Dumont B, Turpin D, Merlin E, Moshous D, Aladjidi N, Neven B, Picard C, Cavazzana M, Fischer A, Durandy A, Stephan JL, Kracker S.

Haematologica. 2017 Jul;102(7):e278-e281. doi: 10.3324/haematol.2017.167601. Epub 2017 Apr 20. No abstract available.

37.

Innovations Needed for Effective Implementation of Ex Vivo Gene Therapies.

Cavazzana M.

Front Med (Lausanne). 2017 Mar 24;4:29. doi: 10.3389/fmed.2017.00029. eCollection 2017. No abstract available.

38.

Gene Therapy for β-Hemoglobinopathies.

Cavazzana M, Antoniani C, Miccio A.

Mol Ther. 2017 May 3;25(5):1142-1154. doi: 10.1016/j.ymthe.2017.03.024. Epub 2017 Apr 1. Review.

39.

INSPIIRED: A Pipeline for Quantitative Analysis of Sites of New DNA Integration in Cellular Genomes.

Sherman E, Nobles C, Berry CC, Six E, Wu Y, Dryga A, Malani N, Male F, Reddy S, Bailey A, Bittinger K, Everett JK, Caccavelli L, Drake MJ, Bates P, Hacein-Bey-Abina S, Cavazzana M, Bushman FD.

Mol Ther Methods Clin Dev. 2016 Dec 18;4:39-49. doi: 10.1016/j.omtm.2016.11.002. eCollection 2017 Mar 17.

40.

INSPIIRED: Quantification and Visualization Tools for Analyzing Integration Site Distributions.

Berry CC, Nobles C, Six E, Wu Y, Malani N, Sherman E, Dryga A, Everett JK, Male F, Bailey A, Bittinger K, Drake MJ, Caccavelli L, Bates P, Hacein-Bey-Abina S, Cavazzana M, Bushman FD.

Mol Ther Methods Clin Dev. 2016 Dec 18;4:17-26. doi: 10.1016/j.omtm.2016.11.003. eCollection 2017 Mar 17.

41.

Reticular dysgenesis: international survey on clinical presentation, transplantation, and outcome.

Hoenig M, Lagresle-Peyrou C, Pannicke U, Notarangelo LD, Porta F, Gennery AR, Slatter M, Cowan MJ, Stepensky P, Al-Mousa H, Al-Zahrani D, Pai SY, Al Herz W, Gaspar HB, Veys P, Oshima K, Imai K, Yabe H, Noroski LM, Wulffraat NM, Sykora KW, Soler-Palacin P, Muramatsu H, Al Hilali M, Moshous D, Debatin KM, Schuetz C, Jacobsen EM, Schulz AS, Schwarz K, Fischer A, Friedrich W, Cavazzana M; European Society for Blood and Marrow Transplantation (EBMT) Inborn Errors Working Party.

Blood. 2017 May 25;129(21):2928-2938. doi: 10.1182/blood-2016-11-745638. Epub 2017 Mar 22.

42.

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

Ginn SL, Hallwirth CV, Liao SH, Teber ET, Arthur JW, Wu J, Lee HC, Tay SS, Hu M, Reddel RR, McCormack MP, Thrasher AJ, Cavazzana M, Alexander SI, Alexander IE.

Mol Ther Nucleic Acids. 2017 Mar 17;6:1-14. doi: 10.1016/j.omtn.2016.11.011. Epub 2016 Dec 10.

43.

Family cord blood banking for sickle cell disease: a twenty-year experience in two dedicated public cord blood banks.

Rafii H, Bernaudin F, Rouard H, Vanneaux V, Ruggeri A, Cavazzana M, Gauthereau V, Stanislas A, Benkerrou M, De Montalembert M, Ferry C, Girot R, Arnaud C, Kamdem A, Gour J, Touboul C, Cras A, Kuentz M, Rieux C, Volt F, Cappelli B, Maio KT, Paviglianiti A, Kenzey C, Larghero J, Gluckman E.

Haematologica. 2017 Jun;102(6):976-983. doi: 10.3324/haematol.2016.163055. Epub 2017 Mar 16.

44.

Cardiac iron overload in chronically transfused patients with thalassemia, sickle cell anemia, or myelodysplastic syndrome.

de Montalembert M, Ribeil JA, Brousse V, Guerci-Bresler A, Stamatoullas A, Vannier JP, Dumesnil C, Lahary A, Touati M, Bouabdallah K, Cavazzana M, Chauzit E, Baptiste A, Lefebvre T, Puy H, Elie C, Karim Z, Ernst O, Rose C.

PLoS One. 2017 Mar 3;12(3):e0172147. doi: 10.1371/journal.pone.0172147. eCollection 2017.

45.

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, Chrétien 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 Mar 2;376(9):848-855. doi: 10.1056/NEJMoa1609677.

46.

Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation.

Gluckman E, Cappelli B, Bernaudin F, Labopin M, Volt F, Carreras J, Pinto Simões B, Ferster A, Dupont S, de la Fuente J, Dalle JH, Zecca M, Walters MC, Krishnamurti L, Bhatia M, Leung K, Yanik G, Kurtzberg J, Dhedin N, Kuentz M, Michel G, Apperley J, Lutz P, Neven B, Bertrand Y, Vannier JP, Ayas M, Cavazzana M, Matthes-Martin S, Rocha V, Elayoubi H, Kenzey C, Bader P, Locatelli F, Ruggeri A, Eapen M; Eurocord, the Pediatric Working Party of the European Society for Blood and Marrow Transplantation, and the Center for International Blood and Marrow Transplant Research.

Blood. 2017 Mar 16;129(11):1548-1556. doi: 10.1182/blood-2016-10-745711. Epub 2016 Dec 13.

47.

Arterio-venous fistula for automated red blood cells exchange in patients with sickle cell disease: Complications and outcomes.

Delville M, Manceau S, Ait Abdallah N, Stolba J, Awad S, Damy T, Gellen B, Sabbah L, Debbache K, Audard V, Beaumont JL, Arnaud C, Chantalat-Auger C, Driss F, Lefrère F, Cavazzana M, Franco G, Galacteros F, Ribeil JA, Gellen-Dautremer J.

Am J Hematol. 2017 Feb;92(2):136-140. doi: 10.1002/ajh.24600. Epub 2016 Dec 7.

48.

Gene-corrected human Munc13-4-deficient CD8+ T cells can efficiently restrict EBV-driven lymphoproliferation in immunodeficient mice.

Soheili T, Rivière J, Ricciardelli I, Durand A, Verhoeyen E, Derrien AC, Lagresle-Peyrou C, de Saint Basile G, Cosset FL, Amrolia P, André-Schmutz I, Cavazzana M.

Blood. 2016 Dec 15;128(24):2859-2862. Epub 2016 Oct 31. No abstract available.

PMID:
27799161
49.

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

Cavazzana M, Ribeil JA, Lagresle-Peyrou C, André-Schmutz I.

Stem Cells Dev. 2017 Jan 15;26(2):71-76. doi: 10.1089/scd.2016.0230. Epub 2016 Oct 16. Review.

50.

Pre-transplant donor CD4- invariant NKT cell expansion capacity predicts the occurrence of acute graft-versus-host disease.

Rubio MT, Bouillié M, Bouazza N, Coman T, Trebeden-Nègre H, Gomez A, Suarez F, Sibon D, Brignier A, Paubelle E, Nguyen-Khoc S, Cavazzana M, Lantz O, Mohty M, Urien S, Hermine O.

Leukemia. 2017 Apr;31(4):903-912. doi: 10.1038/leu.2016.281. Epub 2016 Oct 14.

PMID:
27740636

Supplemental Content

Loading ...
Support Center