Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome von Magnani, A., Semeraro, M., Adam, F., Booth, C., Dupré, L., Morris, E. C., Gabrion, A., Roudaut, C., Borgel, D., Toubert, A., Clave, E., Abdo, C., Gorochov, G., Petermann, R., Guiot, M., Miyara, M., Moshous, D., Magrin, E., Denis, A., Suarez, F., Lagresle, C., Roche, A. M., Everett, J., Trinquand, A., Guisset, M., Bayford, J. Xu, Hacein-Bey-Abina, S., Kauskot, A., Elfeky, R., Rivat, C., Abbas, S., Gaspar, H. B., Macintyre, E., Picard, C., Bushman, F. D., Galy, A., Fischer, A., Six, E., Thrasher, A. J., Cavazzana, M.

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Author Correction: Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome von Magnani, A., Semeraro, M., Adam, F., Booth, C., Dupré, L., Morris, E. C., Gabrion, A., Roudaut, C., Borgel, D., Toubert, A., Clave, E., Abdo, C., Gorochov, G., Petermann, R., Guiot, M., Miyara, M., Moshous, D., Magrin, E., Denis, A., Suarez, F., Lagresle, C., Roche, A. M., Everett, J., Trinquand, A., Guisset, M., Bayford, J. Xu, Hacein-Bey-Abina, S., Kauskot, A., Elfeky, R., Rivat, C., Abbas, S., Gaspar, H. B., Macintyre, E., Picard, C., Bushman, F. D., Galy, A., Fischer, A., Six, E., Thrasher, A. J., Cavazzana, M.

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Non-infectious Lung Disease in Patients with Adenosine Deaminase Deficient Severe Combined Immunodeficiency von Booth, C., Algar, V. E., Xu-Bayford, J., Fairbanks, L., Owens, C., Gaspar, H. B.

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Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency von Kohn, Donald B, Booth, Claire, Shaw, Kit L, Xu-Bayford, Jinhua, Garabedian, Elizabeth, Trevisan, Valentina, Carbonaro-Sarracino, Denise A, Soni, Kajal, Terrazas, Dayna, Snell, Katie, Ikeda, Alan, Leon-Rico, Diego, Moore, Theodore B, Buckland, Karen F, Shah, Ami J, Gilmour, Kimberly C, De Oliveira, Satiro, Rivat, Christine, Crooks, Gay M, Izotova, Natalia, Tse, John, Adams, Stuart, Shupien, Sally, Ricketts, Hilory, Davila, Alejandra, Uzowuru, Chilenwa, Icreverzi, Amalia, Barman, Provaboti, Campo Fernandez, Beatriz, Hollis, Roger P, Coronel, Maritess, Yu, Allen, Chun, Krista M, Casas, Christian E, Zhang, Ruixue, Arduini, Serena, Lynn, Frances, Kudari, Mahesh, Spezzi, Andrea, Zahn, Marco, Heimke, Rene, Labik, Ivan, Parrott, Roberta, Buckley, Rebecca H, Reeves, Lilith, Cornetta, Kenneth, Sokolic, Robert, Hershfield, Michael, Schmidt, Manfred, Candotti, Fabio, Malech, Harry L, Thrasher, Adrian J, Gaspar, H. Bobby

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Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood? von Fernandes, Juliana F., Rocha, Vanderson, Labopin, Myriam, Neven, Benedicte, Moshous, Despina, Gennery, Andrew R., Friedrich, Wilhelm, Porta, Fulvio, Diaz de Heredia, Cristina, Wall, Donna, Bertrand, Yves, Veys, Paul, Slatter, Mary, Schulz, Ansgar, Chan, Ka Wah, Grimley, Michael, Ayas, Mouhab, Gungor, Tayfun, Ebell, Wolfram, Bonfim, Carmem, Kalwak, Krzysztof, Taupin, Pierre, Blanche, Stéphane, Gaspar, H. Bobby, Landais, Paul, Fischer, Alain, Gluckman, Eliane, Cavazzana-Calvo, Marina

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Outcome in patients with severe combined immunodeficiency diagnosed at birth in comparison with proband siblings: the case for neonatal screening von Brown, L, Xu-Bayford, J, Davies, G, Veys, P, Gennery, A, Slatter, M, Gaspar, B

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Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome von Magnani, A, Semeraro, M, Adam, F, Booth, C, Dupré, L, Morris, EC, Gabrion, A, Roudaut, C, Borgel, D, Toubert, A, Clave, E, Abdo, C, Gorochov, G, Petermann, R, Guiot, M, Miyara, M, Moshous, D, Magrin, E, Denis, A, Suarez, F, Lagresle, C, Roche, AM, Everett, J, Trinquand, A, Guisset, M, Bayford, J Xu, Hacein-Bey-Abina, S, Kauskot, A, Elfeky, R, Rivat, C, Abbas, S, Gaspar, HB, Macintyre, E, Picard, C, Bushman, FD, Galy, A, Fischer, A, Six, E, Thrasher, AJ, Cavazzana, M

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Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency von Kohn, DB, Booth, C, Shaw, KL, Xu-Bayford, J, Garabedian, E, Trevisan, V, Carbonaro-Sarracino, DA, Soni, K, Terrazas, D, Snell, K, Ikeda, A, Leon-Rico, D, Moore, TB, Buckland, KF, Shah, AJ, Gilmour, KC, De Oliveira, S, Rivat, C, Crooks, GM, Izotova, N, Tse, J, Adams, S, Shupien, S, Ricketts, H, Davila, A, Uzowuru, C, Icreverzi, A, Barman, P, Campo Fernandez, B, Hollis, RP, Coronel, M, Yu, A, Chun, KM, Casas, CE, Zhang, R, Arduini, S, Lynn, F, Kudari, M, Spezzi, A, Zahn, M, Heimke, R, Labik, I, Parrott, R, Buckley, RH, Reeves, L, Cornetta, K, Sokolic, R, Hershfield, M, Schmidt, M, Candotti, F, Malech, HL, Thrasher, AJ, Gaspar, HB

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Lentiviral gene therapy for X-linked chronic granulomatous disease von Kohn, Donald B., Booth, Claire, Kang, Elizabeth M., Pai, Sung-Yun, Shaw, Kit L., Santilli, Giorgia, Armant, Myriam, Buckland, Karen F., Choi, Uimook, De Ravin, Suk See, Dorsey, Morna J., Kuo, Caroline Y., Leon-Rico, Diego, Rivat, Christine, Izotova, Natalia, Gilmour, Kimberly, Snell, Katie, Dip, Jinhua Xu-Bayford, Darwish, Jinan, Morris, Emma C., Terrazas, Dayna, Wang, Leo D., Bauser, Christopher A., Paprotka, Tobias, Kuhns, Douglas B., Gregg, John, Raymond, Hayley E., Everett, John K., Honnet, Geraldine, Biasco, Luca, Newburger, Peter E., Bushman, Frederic D., Grez, Manuel, Gaspar, H. Bobby, Williams, David A., Malech, Harry L., Galy, Anne, Thrasher, Adrian J.

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Cognitive and behavioral abnormalities in children after hematopoietic stem cell transplantation for severe congenital immunodeficiencies von Titman, Penny, Pink, Elizabeth, Skucek, Emily, O'Hanlon, Katherine, Cole, Tim J., Gaspar, Jane, Xu-Bayford, JinHua, Jones, Alison, Thrasher, Adrian J., Davies, E. Graham, Veys, Paul A., Gaspar, H. Bobby

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Severe Combined Immunodeficiency (SCID)—the Irish Experience von Burns, Heather, Collins, Abigail, Marsden, Paul, Flood, Terence J., Slatter, Mary A., Booth, Claire, Xu-Bayford, Jinhua, Leahy, Timothy Ronan

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Outcomes Following Gene Therapy in Patients With Severe Wiskott-Aldrich Syndrome von Hacein-Bey Abina, Salima, Gaspar, H. Bobby, Blondeau, Johanna, Caccavelli, Laure, Charrier, Sabine, Buckland, Karen, Picard, Capucine, Six, Emmanuelle, Himoudi, Nourredine, Gilmour, Kimberly, McNicol, Anne-Marie, Hara, Havinder, Xu-Bayford, Jinhua, Rivat, Christine, Touzot, Fabien, Mavilio, Fulvio, Lim, Annick, Treluyer, Jean-Marc, Héritier, Sébastien, Lefrère, Francois, Magalon, Jeremy, Pengue-Koyi, Isabelle, Honnet, Géraldine, Blanche, Stéphane, Sherman, Eric A, Male, Frances, Berry, Charles, Malani, Nirav, Bushman, Frederic D, Fischer, Alain, Thrasher, Adrian J, Galy, Anne, Cavazzana, Marina

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Successful Reconstitution of Immunity in ADA-SCID by Stem Cell Gene Therapy Following Cessation of PEG-ADA and Use of Mild Preconditioning von Gaspar, H Bobby, Bjorkegren, Emma, Parsley, Kate, Gilmour, Kimberly C, King, Doug, Sinclair, Joanna, Zhang, Fang, Giannakopoulos, Aris, Adams, Stuart, Fairbanks, Lynette D, Gaspar, Jane, Henderson, Lesley, Xu-Bayford, Jin Hua, Davies, E Graham, Veys, Paul A, Kinnon, Christine, Thrasher, Adrian J

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Lentiviral-mediated Gene Therapy for Patients with Fanconi Anemia [Group A]: Updated Results from Global RP-L102 Clinical Trials von Czechowicz, Agnieszka, Sevilla, Julián, Booth, Claire, Agarwal, Rajni, Zubicaray, Josune, Río, Paula, Navarro, Susana, Chetty, Kritika, O'Toole, Grainne, Xu-Bayford, Jinhua, Ancliff, Philip, Sebastián, Elena, Choi, Grace, Zeini, Miriam, Nicoletti, Eileen, Wagner, John E, Rao, Gayatri R., Thrasher, Adrian J., Schwartz, Jonathan D., Roncarolo, Maria Grazia, Bueren, Juan A.

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Lentiviral Gene Therapy with Low Dose Conditioning for X-Linked SCID Results in Complete Immune Reconstitution and No Evidence of Clonal Expansion von Booth, Claire, Kohn, Donald B., Armant, Myriam, Prockop, Susan, Buckland, Karen, Chandra, Sharat, Chandrakasan, Shanmuganathan, Chetty, Kritika, Dansereau, Colleen, de Oliveira, Satiro, Everett, John, León Rico, Diego, London, Wendy B., Xu-Bayford, Jin Hua, Moore, Theodore B., Nagarsheth, Nisha, Parikh, Suhag, Terrazas, Dayna, White, Shanna L, Schambach, Axel, Bushman, Frederic D., Thrasher, Adrian J., Williams, David A., Pai, Sung-Yun

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Interim Results from an Ongoing Phase 1/2 Study of Lentiviral-mediated Ex-vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I) von Booth, Claire, Sevilla, Julián, Rao, Gayatri R., Chitty Lopez, Maria, Almarza, Elena, Terrazas, Dayna, Zubicaray, Josune, González Vicent, Marta, Chetty, Kritika, O'Toole, Grainne, Xu-Bayford, Jinhua, Nicoletti, Eileen, Fernandes, Augustine, Kuo, Caroline, de Oliveira, Satiro, Moore, Theodore B., Choi, Grace, Zeini, Miriam, Mesa-Núñez, Cristina, Thrasher, Adrian J., Bueren, Juan A., Schwartz, Jonathan D., Kohn, Donald B.

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Bipotent Lymphoid Progenitors Independently Maintain Long-Term Genetically Engineered T and NK Cell Production in Humans von Izotova, Natalia, Rivat, Christine, Baricordi, Cristina, Blanco-Alvarez, Elena, Pellin, Danilo, Watt, Eleanor, Gkazi, Soragia Athina, Adams, Stuart, Gilmour, Kimberly, Bayford, Jinhua Xu, Booth, Claire, Gaspar, Bobby H, Thrasher, Adrian J., Biasco, Luca

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Long-Term Follow-up Study after Lentiviral Hematopoietic Stem/Progenitor Cell Gene Therapy for Wiskott - Aldrich Syndrome von Magnani, Alessandra, Semeraro, Michaela, ADAM, Frédéric, Booth, Claire, Dupre, Loic, Morris, Emma C, Gabrion, Aurélie, Roudaut, Cecile, Borgel, Delphine, Toubert, Antoine, Clave, Emmanuel, Abdo, Chrystelle, Gorochov, Guy, Petermann, Rachel, Guiot, Mélanie, Miyara, Makoto, Moshous, Despina, Magrin, Elisa, Denis, Adeline, Suarez, Felipe, Lagresle-Peyrou, Chantal, Doto, Aoife, Everett, John K., Trinquand, Amélie, Guisset, Marianne, Xu-Bayford, Jin Hua, Hacein-Bey-Abina, Salima, Kauskot, Alexandre, Elfeky, Reem, Rivat, Christine, Abbas, Sarah, Gaspar, Bobby H, Macintyre, Elizabeth A., Picard, Capucine, Bushman, Frederic D., Galy, Anne, Fischer, Alain, Six, Emmanuelle, Thrasher, Adrian J., Cavazzana, Marina

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Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency... von Kohn, Donald B., Shaw, Kit L., Garabedian, Elizabeth, Carbonaro-Sarracino, Denise Ann, Moore, Theodore B., De Oliveira, Satiro N., Crooks, Gay M., Tse, John, Shupien, Sally, Terrazas, Dayna, Davila, Alejandra, Icreverzi, Amalia, Yu, Allen, Chun, Krista M., Casas, Christian E., Barman, Provaboti, Coronel, Maritess, Campo Fernandez, Beatriz, Zhang, Ruixue, Hollis, Roger P., Uzowuru, Chilenwa, Ricketts, Hilory, Bayford, Jinhua Xu, Trevisan, Valentina, Arduini, Serena, Lynn, Frances, Kudari, Mahesh, Spezzi, Andrea, Reeves, Lilith, Cornetta, Kenneth, Sokolic, Robert A., Parrott, Roberta, Buckley, Rebecca, Booth, Claire, Candotti, Fabio, Malech, Harry L., Thrasher, Adrian J., Gaspar, H. Bobby

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Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results from an Ongoing Phase 1/2 Study von Booth, Claire, Sevilla, Julian, Rao, Gayatri, Lopez, Maria Chitty, Almarza, Elena, Terrazas, Dayna, Zubicaray, Josune, Gonzalez-Vicent, Marta, Chetty, Kritika, Xu-Bayford, Jinhua, O’Toole, Grainne, Fernandes, Augustine, Kuo, Caroline Y., De Oliveira, Satiro N., Mesa-Núñez, Cristina, Moore, Theodore B., Nicoletti, Eileen, Choi, Grace, Zeini, Miriam, Thrasher, Adrian J., Bueren, Juan, Schwartz, Jonathan, Kohn, Donald B.

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