Cell-pid 2011 & 2012 publications

LIBRARY
2011 Publications

Abeyewickreme, A., Thrasher, A.J., & Kinnon, C. 2011. Bone morphogenetic protein-4 (BMP4) up
regulates key haematopoietic genes in differentiating embryonic stem cells treated with BMP4 short
hairpin RNA. Br.J.Haematol. Epub.
Almarza, E., Zhang, F., Santilli, G., Blundell, M.P., Howe, S.J., Thornhill, S.I., Bueren, J.A., & Thrasher, A.J. 2011. Correction of SCID-X1 Using an Enhancerless Vav Promoter. Hum.Gene Ther., 22, (3) 263-270 Bacchelli, C., Buckland, K.F., Buckridge, S., Salzer, U., Schneider, P., Thrasher, A.J., & Gaspar, H.B. 2011. The C76R transmembrane activator and calcium modulator cyclophilin ligand interactor mutation disrupts antibody production and B-cell homeostasis in heterozygous and homozygous mice. J.Allergy Clin.Immunol., 127, (5) 1253-1259 Bartholomae, C.C., Arens, A., Balaggan, K.S., Yanez-Munoz, R.J., Montini, E., Howe, S.J., Paruzynski, A., Korn, B., Appelt, J.U., Macneil, A., Cesana, D., Abel, U., Glimm, H., Naldini, L., Ali, R.R., Thrasher, A.J., von, K.C., & Schmidt, M. 2011. Lentiviral vector integration profiles differ in rodent postmitotic tissues. Mol.Ther., 19, (4) 703-710 Bianchi M, Niemiec MJ, Siler U, Urban CF, Reichenbach J. (2011) Restoration of anti-Aspergillus defense by neutrophil extracellular traps in human chronic granulomatous disease after gene therapy is calprotectin-dependent J Allergy Clin Immunol. May;127(5):1243-52.e7. Biasco L, Baricordi C, Aiuti A. (2011) Retroviral integrations in gene therapy trials. Revised version submitted to Mol Therapy. Blundell, M.P., Worth, A., Bouma, G., & Thrasher, A.J. 2011. The Wiskott-Aldrich syndrome: The actin cytoskeleton and immune cell function. Dis.Markers, 29, (3) 157-175 Booth, C., Gilmour, K.C., Veys, P., Gennery, A.R., Slatter, M.A., Chapel, H., Heath, P.T., Steward, C.G., Smith, O., O'Meara, A., Kerrigan, H., Mahlaoui, N., Cavazzana-Calvo, M., Fischer, A., Moshous, D., Blanche, S., Pachlopnick-Schmid, J., Latour, S., de Saint-Basile, G., Albert, M., Notheis, G., Rieber, N., Strahm, B., Ritterbusch, H., Lankester, A., Hartwig, N.G., Meyts, I., Plebani, A., Soresina, A., Finocchi, A., Pignata, C., Cirillo, E., Bonanomi, S., Peters, C., Kalwak, K., Pasic, S., Sedlacek, P., Jazbec, J., Kanegane, H., Nichols, K.E., Hanson, I.C., Kapoor, N., Haddad, E., Cowan, M., Choo, S., Smart, J., Arkwright, P.D., & Gaspar, H.B. 2011. X-linked lymphoproliferative disease due to SAP/SH2D1A deficiency: a multicenter study on the manifestations, management and outcome of the disease. Blood, 117, (1) 53-62 Bouma, G., Ancliff, P.J., Thrasher, A.J., & Burns, S.O. 2010. Recent advances in the understanding of genetic defects of neutrophil number and function. Br.J.Haematol., 151, (4) 312-326 Bouma, G., Mendoza-Naranjo, A., Blundell, M.P., de, F.E., Parsley, K.L., Burns, S.O., & Thrasher, A.J. 2011. Cytoskeletal remodeling mediated by WASp in dendritic cells is necessary for normal immune synapse formation and T-cell priming. Blood, 118, (9) 2492-2501 C Brendel, U Mu¨ ller-Kuller, S Schultze-Strasser1, S Stein1, L Chen-Wichmann, A Krattenmacher, H Kunkel, A Dillmann, MN Antoniou3and M Grez 2011. Physiological regulation of transgene expression by a lentiviral vector containing the A2UCOE linked to a myeloid promoter, Gene Therapy, 1-12 Brown, L., Xu-Bayford, J., Allwood, Z., Slatter, M., Cant, A., Davies, E.G., Veys, P., Gennery, A.R., & Gaspar, H.B. 2011. Neonatal diagnosis of severe combined immunodeficiency leads to significantly improved survival outcome: the case for newborn screening. Blood, 117, (11) 3243-3246 Cancrini C, Scarselli A, Scaramuzza S, Chiriaco M, Di Cesare S, Di Matteo G, Romiti ML, Palma P, De Felice L, Palumbo G, Pinto RM, De Vito R, Racioppi L, Livadiotti S, Fischer A, Rossi P, Caniglia M, Aiuti A. (2011) Early-onset monocyte-B-natural killer-dendritic cells’ deficiency successfully treated with hematopoietic stem cell transplantation. J Allergy Clin Immunol. 128, 897-900.e1. Cole, T.S., Jones, L.K., McGrogan, P., Pearce, M.S., Flood, T.J., Cant, A.J., Goldblatt, D., Thrasher, A.J., Gennery, A.R., McKendrick, F., & Titman, P. 2011. Emotional and behavioural difficulties in chronic granulomatous disease. Arch.Dis.Child Deichmann A, Brugman MH, Bartholomae CC, Schwarzwaelder K, Verstegen MM, Howe SJ, Arens A, Ott MG, Hoelzer D, Seger R, Grez M, Hacein-Bey-Abina S, Cavazzana-Calvo M, Fischer A, Paruzynski A, Gabriel R, Glimm H, Abel U, Cattoglio C, Mavilio F, Cassani B, Aiuti A, Dunbar CE, Baum C, Gaspar HB, Thrasher AJ, von Kalle C, Schmidt M, Wagemaker G. Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy. Mol Ther. 2011 Nov;19(11):2031-9. Epub 2011 Aug 23. Deichmann, A., Brugman, M.H., Bartholomae, C.C., Schwarzwaelder, K., Verstegen, M.M., Howe, S.J., Arens, A., Ott, M.G., Hoelzer, D., Seger, R., Grez, M., Hacein-Bey-Abina, S., Cavazzana-Calvo, M., Fischer, A., Paruzynski, A., Gabriel, R., Glimm, H., Abel, U., Cattoglio, C., Mavilio, F., Cassani, B., Aiuti, A., Dunbar, C.E., Baum, C., Gaspar, H.B., Thrasher, A.J., von, K.C., Schmidt, M., & Wagemaker, G. 2011. Insertion Sites in Engrafted Cells Cluster Within a Limited Repertoire of Genomic Areas After Gammaretroviral Vector Gene Therapy. Mol.Ther. Epub. Di, W.L., Larcher, F., Semenova, E., Talbot, G.E., Harper, J.I., Del, R.M., Thrasher, A.J., & Qasim, W. 2011. Ex-vivo Gene Therapy Restores LEKTI Activity and Corrects the Architecture of Netherton Syndrome-derived Skin Grafts. Mol Ther, 19, (2) 408-416 Di, W.L., Semenova, E., Larcher, F., Del, R.M., Harper, J.I., Thrasher, A., & Qasim, W. 2011. Human involucrin promoter mediates repression resistant and compartment specific LEKTI expression. Hum.Gene Ther. Epub. Galy, A. & Thrasher, A.J. 2011. Gene therapy for the Wiskott-Aldrich syndrome. Curr.Opin.Allergy Clin.Immunol., 11, (6) 545-550 Gaspar, H.B. 2011. Induced pluripotent stem cells and primary immunodeficiencies: a new frontier reached, a new world beyond? J.Allergy Clin.Immunol., 127, (6) 1408-1409 Gaspar, H.B., Cooray, S., Gilmour, K.C., Parsley, K.L., Zhang, F., Adams, S., Bjorkegren, E., Bayford, J., Brown, L., Davies, E.G., Veys, P., Fairbanks, L., Bordon, V., Petropolou, T., Kinnon, C., & Thrasher, A.J. 2011. Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction. Sci.Transl.Med., 3, (97) 97ra80 Gaspar, H.B., Cooray, S., Gilmour, K.C., Parsley, K.L., Adams, S., Howe, S.J., Al, G.A., Bayford, J., Brown, L., Davies, E.G., Kinnon, C., & Thrasher, A.J. 2011. Long-term persistence of a polyclonal T cell repertoire after gene therapy for x-linked severe combined immunodeficiency. Sci.Transl.Med., 3, (97) 97ra79 Georgiadis, A., Tschernutter, M., Bainbridge, J.W., Balaggan, K.S., Mowat, F., West, E.L., Munro, P.M., Thrasher, A.J., Matter, K., Balda, M.S., & Ali, R.R. 2010. The Tight Junction Associated Signalling Proteins ZO-1 and ZONAB Regulate Retinal Pigment Epithelium Homeostasis in Mice. PLoS.One., 5, (12) e15730 Gholam, C., Grigoriadou, S., Gilmour, K.C., & Gaspar, H.B. 2011. Familial haemophagocytic lymphohistiocytosis: advances in the genetic basis, diagnosis and management. Clin.Exp.Immunol., 163, (3) 271-283 Golan K, Vagima Y, Ludin A, Itkin T, Cohen-Gur S, Kalinkovich A, Kollet O, Kim C, Schajnovitz A, Ovadya Y, Lapid K, Shivtiel S, Morris A, Ratajczak M, & Lapidot T. (2012). S1P promotes murine progenitor cell egress and mobilization via S1P1 mediated ROS signalling and SDF-1 release. Blood. 119(11): 2478-2488. Grez, M., Reichenbach, J., Schwable, J., Seger, R., Dinauer, M.C., & Thrasher, A.J. 2011. Gene therapy of chronic granulomatous disease: the engraftment dilemma. Mol Ther, 19, (1) 28-35 Huston MW, van Til NP, Visser TP, Arshad S, Brugman MH, Cattoglio C, Nowrouzi A, Li Y, Schambach A, Schmidt M, Baum C, von Kalle C, Mavilio F, Zhang F, Blundell MP, Thrasher AJ, Verstegen MM, Wagemaker G. Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioning. Mol Ther. 2011 Oct;19(10):1867-77. Epub 2011 Jul 12. Huston, M.W., van Til, N.P., Visser, T.P., Arshad, S., Brugman, M.H., Cattoglio, C., Nowrouzi, A., Li, Y., Schambach, A., Schmidt, M., Baum, C., von, K.C., Mavilio, F., Zhang, F., Blundell, M.P., Thrasher, A.J., Verstegen, M.M., & Wagemaker, G. 2011. Correction of Murine SCID-X1 by Lentiviral Gene Therapy Using a Codon-optimized IL2RG Gene and Minimal Pretransplant Conditioning. Mol.Ther., 19, (10) 1867-1877 Ian C.D. Johnston, Constanze Lehmann, Martin Biehl, Melanie Fahrendorff, Volker Huppert, and Stefan Miltenyi (2011). Fully automated cell selection, cultivation, and genetic modification in a closed environment for the flexible manufacturing of cellular products. Mol. Therapy 19: S227 Kane, N.M., Nowrouzi, A., Mukherjee, S., Blundell, M.P., Greig, J.A., Lee, W.K., Houslay, M.D., Milligan, G., Mountford, J.C., von, K.C., Schmidt, M., Thrasher, A.J., & Baker, A.H. 2010. Lentivirus-mediated Reprogramming of Somatic Cells in the Absence of Transgenic Transcription Factors. Mol Ther, 18, (12) 2139-2145 Mandegar, M.A., Moralli, D., Khoja, S., Cowley, S., Chan, D.Y., Yusuf, M., Mukherjee, S., Blundell, M.P., Volpi, E.V., Thrasher, A.J., James, W., & Monaco, Z.L. 2011. Functional human artificial chromosomes are generated and stably maintained in human embryonic stem cells. Hum.Mol.Genet., 20, (15) 2905-2913 Matteo Bianchi, MSc,a* Maria J. Niemiec, MSc,b* Ulrich Siler, PhD,a Constantin F. Urban, PhD,b_and Janine Reichenbach, MDa_ Zurich, Switzerland, and Ume_a, Sweden, Mendoza-Naranjo, A., Bouma, G., Pereda, C., Ramirez, M., Webb, K.F., Tittarelli, A., Lopez, M.N., Kalergis, A.M., Thrasher, A.J., Becker, D.L., & Salazar-Onfray, F. 2011. Functional gap junctions accumulate at the immunological synapse and contribute to T cell activation. J.Immunol., 187, (6) 3121-3132 Metelo, J., Ward, N., Thrasher, A.J., & Burns, S.O. 2011. Lentivectors are efficient tools to manipulate the dendritic cell cytoskeleton. Cytoskeleton (Hoboken.), 68, (8) 434-445 Monypenny, J., Chou, H.C., Banon-Rodriguez, I., Thrasher, A.J., Anton, I.M., Jones, G.E., & Calle, Y. 2011. Role of WASP in cell polarity and podosome dynamics of myeloid cells. Eur.J.Cell Biol., 90, (2-3) 198-204 Moratto, D., Giliani, S., Bonfim, C., Mazzolari, E., Fischer, A., Ochs, H.D., Cant, A.J., Thrasher, A.J., Cowan, M.J., Albert, M.H., Small, T., Pai, S.Y., Haddad, E., Lisa, A., Hambleton, S., Slatter, M., Cavazzana-Calvo, M., Mahlaoui, N., Picard, C., Torgerson, T.R., Burroughs, L., Koliski, A., Neto, J.Z., Porta, F., Qasim, W., Veys, P., Kavanau, K., Honig, M., Schulz, A., Friedrich, W., & Notarangelo, L.D. 2011. Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study. Blood, 118, (6) 1675-1684 Mukherjee, S. & Thrasher, A.J. 2011. iPSCs: Unstable Origins? Mol.Ther., 19, (7) 1188-1190 Mukherjee, S. & Thrasher, A.J. 2011. Progress and prospects: advancements in retroviral vector design, generation, and application. Hum.Gene Ther., 22, (10) 1171-1174 Mukherjee, S., Santilli, G., Blundell, M.P., Navarro, S., Bueren, J.A., & Thrasher, A.J. 2011. Generation of functional neutrophils from a mouse model of x-linked chronic granulomatous disorder using induced pluripotent stem cells. PLoS.One., 6, (3) e17565 Niek P. van Til, Helen de Boer, Nomusa Mashamba, Aga Wabik, Marshall Huston, Roya Sarwari, Trudi P. Visser, Elena Fontana, Pietro Luigi Poliani, Barbara Cassani, Fang Zhang6, Adrian J Thrasher, Anna Villa, and Gerard Wagemaker1. Codon-optimization of RAG2 improves immune function by lentiviral gene therapy of murine Rag2 severe combined immunodeficiency. Revision submitted Picard, C., von, B.H., Ghandil, P., Chrabieh, M., Levy, O., Arkwright, P.D., McDonald, D., Geha, R.S., Takada, H., Krause, J.C., Creech, C.B., Ku, C.L., Ehl, S., Marodi, L., Al-Muhsen, S., Al-Hajjar, S., Al-Ghonaium, A., Day-Good, N.K., Holland, S.M., Gallin, J.I., Chapel, H., Speert, D.P., Rodriguez-Gallego, C., Colino, E., Garty, B.Z., Roifman, C., Hara, T., Yoshikawa, H., Nonoyama, S., Domachowske, J., Issekutz, A.C., Tang, M., Smart, J., Zitnik, S.E., Hoarau, C., Kumararatne, D.S., Thrasher, A.J., Davies, E.G., Bethune, C., Sirvent, N., de, R.D., Camcioglu, Y., Vasconcelos, J., Guedes, M., Vitor, A.B., Rodrigo, C., Almazan, F., Mendez, M., Arostegui, J.I., Alsina, L., Fortuny, C., Reichenbach, J., Verbsky, J.W., Bossuyt, X., Doffinger, R., Abel, L., Puel, A., & Casanova, J.L. 2010. Clinical Features and Outcome of Patients With IRAK-4 and MyD88 Deficiency. Medicine (Baltimore), 89, (6) 403-425 Pike-Overzet, K., Rodijk, M., Ng, Y.Y., Baert, M.R., Lagresle-Peyrou, C., Schambach, A., Zhang, F., Hoeben, R.C., Hacein-Bey-Abina, S., Lankester, A.C., Bredius, R.G., Driessen, G.J., Thrasher, A.J., Baum, C., Cavazzana-Calvo, M., van Dongen, J.J., & Staal, F.J. 2011. Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer. Leukemia, 25, (9) 1471-1483 Qasim, W., Derniame, S., Gilmour, K., Chiesa, R., Weber, M., Adams, S., Rao, K., Amrolia, P., Goulden, N., Veys, P., & Gaspar, H. 2011. Third-party virus-specific T cells eradicate adenoviraemia but trigger bystander graft-versus-host disease. Br.J.Haematol., 154, (1) 150-153 Sánchez-Antequera Y, Mykhaylyk O, Cengizeroglu A, van Til NP, Anton M, Johnston ICD, Pojda Z, Wagemaker G, Plank C (2011). Magselectofection: An integrated method of nanomagnetic separation and genetic modification of target cells. Blood 117: e171–181 Santilli, G., Almarza, E., Brendel, C., Choi, U., Beilin, C., Blundell, M.P., Haria, S., Parsley, K.L., Kinnon, C., Malech, H.L., Bueren, J.A., Grez, M., & Thrasher, A.J. 2011. Biochemical Correction of X-CGD by a Novel Chimeric Promoter Regulating High Levels of Transgene Expression in Myeloid Cells. Mol Ther, 19, (1) 122-132 Sauer AV, Brigida I, Carriglio N, Jofra Hernandez R, Scaramuzza S, Clavenna D, Sanvito F, Poliani PL, Gagliani N, Carlucci N, Tabucchi A, Roncarolo MG, Traggiai E, Villa A, Aiuti A. Alterations in the adenosine metabolism and CD39/CD73 adenosinergic machinery cause loss of Treg cell function and autoimmunity in ADA-deficient SCID. Revised version submitted to Blood Journal. Sauer AV, Morbach H, Brigida I, Ng YS, Aiuti A, Meffre E. Defective B cell tolerance due to adenosine deaminase deficiency is corrected by gene therapy. Submitted to Journal of Clinical Investigation. Scaramuzza S, Biasco L, Ripamonti A, Castiello MC, Loperfido M, Draghici E, Jofra Hernandez R, Benedicenti F, Radrizzani M, Salomoni M, Ranzani M, Bartholomae CC, Vicenzi E, Finocchi A, Bredius R, Bosticardo M, Schmidt M, von Kalle C, Montini E, Biffi A, Roncarolo MG, Naldini L, Villa A, Aiuti A (2011) Preclinical safety and efficacy of human CD34+ cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich Syndrome. Revised version submitted to Mol Therapy Schajnovitz A, Itkin T, D'Uva G, Kalinkovich A, Golan K, Ludin A, Cohen D, Shulman Z, Avigdor A, Nagler A, Kollet O, Seger R, Lapidot T. CXCL12 secretion by bone marrow stromal cells is dependent on cell contact and mediated by connexin-43 and connexin-45 gap junctions. Nat Immunol. 2011 May;12(5):391-8. Selleri S, Brigida I, Casiraghi M, Scaramuzza S, Cappelli B, Cassani B, Ferrua F, Aker M, Slavin S, Scarselli A, Cancrini C, Marktel S, Roncarolo MG and Aiuti A. (2011) In vivo T-cell dynamics during immune reconstitution after hematopoietic stem cell gene therapy in adenosine deaminase severe combined immune deficiency. J Allergy Clin Immunol. 127, 1368-1375.e8. Shaw, S.W., Bollini, S., Nader, K.A., Gastadello, A., Mehta, V., Filppi, E., Cananzi, M., Gaspar, H.B., Qasim, W., De, C.P., & David, A.L. 2010. Autologous transplantation of amniotic fluid derived mesenchymal stem cells into sheep fetuses. Cell Transplant. Epub Skucek, E., Butler, S., Gaspar, H.B., & Titman, P. 2011. Social outcome in children treated by haematopoietic cell transplant for congenital immunodeficiency. Bone Marrow Transplant. Epub. Slatter, M.A., Rao, K., Amrolia, P., Flood, T., Abinun, M., Hambleton, S., Nademi, Z., Goulden, N., Davies, G., Qasim, W., Gaspar, H.B., Cant, A., Gennery, A.R., & Veys, P. 2011. Treosulfan-based conditioning regimens for hematopoietic stem cell transplantation in children with primary immunodeficiency: United Kingdom experience. Blood, 117, (16) 4367-4375 50 Thrasher, A.J. & Segal, A.W. 2011. A phagocyte dilemma. Nat.Immunol., 12, (3) 201-202 Tolar, J., Adair, J.E., Antoniou, M., Bartholomae, C.C., Becker, P.S., Blazar, B.R., Bueren, J., Carroll, T., Cavazzana-Calvo, M., Clapp, D.W., Dalgleish, R., Galy, A., Gaspar, H.B., Hanenberg, H., von, K.C., Kiem, H.P., Lindeman, D., Naldini, L., Navarro, S., Renella, R., Rio, P., Sevilla, J., Schmidt, M., Verhoeyen, E., Wagner, J.E., Williams, D.A., & Thrasher, A.J. 2011. Stem cell gene therapy for fanconi anemia: report from the 1st international fanconi anemia gene therapy working group meeting. Mol.Ther., 19, (7) 1193-1198 Van der Loo, J.C., Swaney, W.P., Grassman, E., Terwilliger, A., Higashimoto, T., Schambach, A., Baum, C., Thrasher, A.J., Williams, D.A., Nordling, D.L., Reeves, L., & Malik, P. 2011. Scale-up and manufacturing of clinical-grade self-inactivating gamma-retroviral vectors by transient transfection. Gene Ther. Epub Ward, N.J., Buckley, S.M., Waddington, S.N., Vandendriessche, T., Chuah, M.K., Nathwani, A.C., McIntosh, J., Tuddenham, E.G., Kinnon, C., Thrasher, A.J., & McVey, J.H. 2011. Codon optimization of human factor VIII cDNAs leads to high-level expression. Blood, 117, (3) 798-807 Williams, D.A. & Thrasher, A.J. 2011. Out of harm's way. Nat.Biotechnol., 29, (1) 41-42 Williams, D.A., Thrasher, A.J., & Baum, C. 2010. Transatlantic consortium spotlights need for changes in gene therapy trials. Mol Ther, 18, (11) 1892 2012 Publications
Aiuti A., Bacchetta R., Seger R., Villa A., Cavazzana-Calvo M. (2012) Gene therapy for primary immunodeficiencies: Part 2 Curr. Opin. Immunol., 24, 585-591. Catucci M., Prete F., Bosticardo M., Castiello M.C., Draghici E., Locci M., Roncarolo M.G., Aiuti A., Benvenuti F., Villa A. (2012) Dendritic cell functional improvement in a preclinical model of lentiviral mediated gene therapy for Wiskott-Aldrich syndrome. Gene Ther., 19, 1150-59, [Epub 2011 Dec 22]. Barzaghi F, Passerini L, Gambineri E, Ciullini Mannurita S, Cornu T, Kang ES, Choe YH, Cancrini C, Corrente S, Ciccocioppo R, Cecconi M, Zuin G, Discepolo V, Sartirana C, Schmidtko J, Ikinciogullari A, Ambrosi A, Roncarolo MG, Olek S, Bacchetta R. Demethylation analysis of the FOXP3 locus shows quantitative defects of regulatory T cells in Ipex-like syndrome J Autoimmun. 2012 Feb;38:49-58. Barzaghi F, Passerini L, Gambineri E, Ciullini Mannurita S, Cornu T, Kang ES, Choe YH, Cancrini C, Corrente S, Ciccocioppo R, Cecconi M, Zuin G, Discepolo V, Sartirana C, Schmidtko J, Ikinciogullari A, Ambrosi A, Roncarolo MG, Olek S, Bacchetta R., J Autoimmun (2012) Demethylation analysis of the FOXP3 locus shows quantitative defects of regulatory T cells in IPEXlike syndrome Feb;38(1):49 58. doi: 10.1016/j.jaut.2011.12.009. Epub 2012 Jan 20. Barzaghi F, Passerini L, Bacchetta R. (2012) Immune dysregulation, polyendocrinopathy, enteropathy, x-linked syndrome: a paradigm of immunodeficiency with autoimmunity. Front Immunol. 2012;3:211. doi: 10.3389/fimmu.2012.00211. Epub 2012 Jul 31. Barroeta Seijas AB, Graziani S, Cancrini C, Finocchi A, Ferrari S, Miniero R, Conti F, Zuntini R, Chini L, Chiarello P, Bengala M, Rossi P, Moschese V, Di Matteo G. The impact of TACI mutations: from hypogammaglobulinemia in infancy to autoimmunity in adulthood. Int J Immunopathol Pharmacol. 2012 Apr-Jun;25(2):407-14. Baum C (editorial). Conditional negative selection of gene-modified hematopoietic stem cells. Molecular Therapy 2012, 20:1841-1842. Biasco L., Baricordi C., Aiuti A. (2012) Retroviral integrations in gene therapy trials. Mol. Ther. 20, 709-16. Brendel C, Müller-Kuller U, Schultze-Strasser S, Stein S, Chen-Wichmann L, Krattenmacher A, Kunkel H, Dillmann A, Antoniou MN, Grez M. Physiological regulation of transgene expression by a lentiviral vector containing the A2UCOE linked to a myeloid promoter. Gene Ther. 2012 Oct;19(10):1018-29 Cavazzana-Calvo M., Fischer A., Hacein-Bey-Abina S., Aiuti A. (2012) Gene of therapy for primary immunodeficiencies: Part 1 Curr. Opin. Immunol., 24, 580-584. Corrigan-Curay J., Cohen-Haguenauer O., O’Reilly M., Ross S.R., Fan H., Rosenberg N., Somia N., King N., Friedmann T., Dunbar C., Aiuti A., Naldini L., Baum C., von Kalle C., Kiem H.P., Montini E., Bushman F., Sorrentino B.P., Carrondo M., Malech H., Gahrton G., Shapiro R., Wolff L., Rosenthal E., Jambou R., Zaia J., Kohn D.B. (2012) Challenges in Vector and Trial Design using Retroviral vectors for Long Term Gene Correction in Hematopoietic Stem Cell Gene Therapy: Summary of aSymposium Sponsored by the NIH Office of Biotechnology Activities and the EC DG-research NoE for the Advancement of Clinical Gene Transfer and Therapy. Mol Ther. 20, 1084-94. Deichmann A, Brugman MH, Bartholomae CC, Schwarzwaelder K, Verstegen MM, Howe SJ, Arens A, Ott MG, Hoelzer D, Seger R, Grez M, Hacein-Bey-Abina S, Cavazzana-Calvo M, Fischer A, Paruzynski A, Gabriel R, Glimm H, Abel U, Cattoglio C, Mavilio F, Cassani B, Aiuti A, Dunbar CE, Baum C, Gaspar HB, Thrasher AJ, von Kalle C, Schmidt M, Wagemaker G. Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy. Mol Ther. 2011 Nov;19(11):2031-9 Golan K, Vagima Y, Ludin A, Itkin T, Cohen-Gur S, Kalinkovich A, Kollet O, Kim C, Schajnovitz A, Ovadya Y, Lapid K, Shivtiel S, Morris A, Ratajczak M, & Lapidot T. (2012). S1P promotes murine progenitor cell egress and mobilization via S1P1 mediated ROS signalling and SDF-1 release. Blood. 119(11): 2478-2488. Hassan A., Booth C., Brightwell A., Allwood Z., Veys P., Rao K., Hoenig M., Friedrich W., Gennery A.R., Slatter M., Bredius R., Finocchi A., Cancrini C., Aiuti A., Lanfranchi A., Porta F., Ridella M., Graham Steward C., Filipovich A., Marsh R., Bordon V., Al-Muhsen S., Al-Mousa H., Alsum Z., Al Dhekri H., Al Ghonaium A., Speckmann C., Fischer A., Mahlaoui N., Nichols K. E., Grunebaum E., Al Zaharani D., Roifman C., Boelens J.J, Davies E. G., Cavazzana-Calvo M., Notarangelo L:, Gaspar H.B. 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