Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency von Reinhardt, Bryanna, Habib, Omar, Shaw, Kit L., Garabedian, Elizabeth, Carbonaro-Sarracino, Denise A., Terrazas, Dayna, Fernandez, Beatriz Campo, De Oliveira, Satiro, Moore, Theodore B., Ikeda, Alan K., Engel, Barbara C., Podsakoff, Gregory M., Hollis, Roger P., Fernandes, Augustine, Jackson, Connie, Shupien, Sally, Mishra, Suparna, Davila, Alejandra, Mottahedeh, Jack, Vitomirov, Andrej, Meng, Wenzhao, Rosenfeld, Aaron M., Roche, Aoife M., Hokama, Pascha, Reddy, Shantan, Everett, John, Wang, Xiaoyan, Luning Prak, Eline T., Cornetta, Kenneth, Hershfield, Michael S., Sokolic, Robert, De Ravin, Suk See, Malech, Harry L., Bushman, Frederic D., Candotti, Fabio, Kohn, Donald B.

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Clinical efficacy of gene-modified stem cells in adenosine deaminase-deficient immunodeficiency von Shaw, Kit L, Garabedian, Elizabeth, Mishra, Suparna, Barman, Provaboti, Davila, Alejandra, Carbonaro, Denise, Shupien, Sally, Silvin, Christopher, Geiger, Sabine, Nowicki, Barbara, Smogorzewska, E Monika, Brown, Berkley, Wang, Xiaoyan, de Oliveira, Satiro, Choi, Yeong, Ikeda, Alan, Terrazas, Dayna, Fu, Pei-Yu, Yu, Allen, Fernandez, Beatriz Campo, Cooper, Aaron R, Engel, Barbara, Podsakoff, Greg, Balamurugan, Arumugam, Anderson, Stacie, Muul, Linda, Jagadeesh, G Jayashree, Kapoor, Neena, Tse, John, Moore, Theodore B, Purdy, Ken, Rishi, Radha, Mohan, Kathey, Skoda-Smith, Suzanne, Buchbinder, David, Abraham, Roshini S, Scharenberg, Andrew, Yang, Otto O, Cornetta, Kenneth, Gjertson, David, Hershfield, Michael, Sokolic, Rob, Candotti, Fabio, Kohn, Donald B

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Cultivated autologous limbal epithelial cell (CALEC) transplantation: Development of manufacturing process and clinical evaluation of feasibility and safety von Jurkunas, Ula V, Yin, Jia, Johns, Lynette K, Li, Sanming, Negre, Helene, Shaw, Kit L, Samarakoon, Lassana, Ayala, Allison R, Kheirkhah, Ahmad, Katikireddy, Kishore, Gauthier, Alex, Ong Tone, Stephan, Kaufman, Aaron R, Ellender, Stacey, Hernandez Rodriguez, Diego E, Daley, Heather, Dana, Reza, Armant, Myriam, Ritz, Jerome

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Foamy Virus Envelope Glycoprotein Is Sufficient for Particle Budding and Release von Shaw, Kit L, Lindemann, Dirk, Mulligan, Mark J, Goepfert, Paul A

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Busulfan Pharmacokinetics in Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Gene Therapy von Bradford, Kathryn L., Liu, Siyu, Krajinovic, Maja, Ansari, Marc, Garabedian, Elizabeth, Tse, John, Wang, Xiaoyan, Shaw, Kit L., Gaspar, H. Bobby, Candotti, Fabio, Kohn, Donald B.

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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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A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency von 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, Laure, Cros, Guilhem, De Oliveira, Satiro, Fernández, Karen S, Guo, Dongjing, Harris, Chad E, Hopkins, Gregory, Lehmann, Leslie E, Lim, Annick, London, Wendy B, van der Loo, Johannes C.M, Malani, Nirav, Male, Frances, Malik, Punam, Marinovic, M. Angélica, McNicol, Anne-Marie, Moshous, Despina, Neven, Benedicte, Oleastro, Matías, Picard, Capucine, Ritz, Jerome, Rivat, Christine, Schambach, Axel, Shaw, Kit L, Sherman, Eric A, Silberstein, Leslie E, Six, Emmanuelle, Touzot, Fabien, Tsytsykova, Alla, Xu-Bayford, Jinhua, Baum, Christopher, Bushman, Frederic D, Fischer, Alain, Kohn, Donald B, Filipovich, Alexandra H, Notarangelo, Luigi D, Cavazzana, Marina, Williams, David A, Thrasher, Adrian J

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369. Development of an Amplifiable Gene Expression System in Lentivirus Vectors von Shaw, Kit L., Pepper, Karen, Petersen, Denise, Ge, Shundi, Crooks, Gay, Kohn, Donald B.

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Gene therapy for adenosine deaminase–deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans von Candotti, Fabio, Shaw, Kit L., Muul, Linda, Carbonaro, Denise, Sokolic, Robert, Choi, Christopher, Schurman, Shepherd H., Garabedian, Elizabeth, Kesserwan, Chimene, Jagadeesh, G. Jayashree, Fu, Pei-Yu, Gschweng, Eric, Cooper, Aaron, Tisdale, John F., Weinberg, Kenneth I., Crooks, Gay M., Kapoor, Neena, Shah, Ami, Abdel-Azim, Hisham, Yu, Xiao-Jin, Smogorzewska, Monika, Wayne, Alan S., Rosenblatt, Howard M., Davis, Carla M., Hanson, Celine, Rishi, Radha G., Wang, Xiaoyan, Gjertson, David, Yang, Otto O., Balamurugan, Arumugam, Bauer, Gerhard, Ireland, Joanna A., Engel, Barbara C., Podsakoff, Gregory M., Hershfield, Michael S., Blaese, R. Michael, Parkman, Robertson, Kohn, Donald B.

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Induction of Fetal Hemoglobin and Reduction of Clinical Manifestations in Patients with Severe Sickle Cell Disease Treated with Shmir-Based Lentiviral Gene Therapy for Post-Transcr... von Esrick, Erica B., Federico, Amy, Abriss, Daniela, Armant, Myriam, Boardman, Kari, Brendel, Christian, Ciuculescu, Marioara-Felicia, Daley, Heather, Dansereau, Colleen, Fernandes, Augustine, Heeney, Matthew M., Justus, David G., Kao, Pei-Chi, Kim, Annette S., Kohn, Donald B., Lehmann, Leslie E., Liu, Donghui, London, Wendy B., Manis, John P, Moore, Theodore B., Morris, Emily, Pellin, Danilo, Proeung, Ellen, Richard, Shanna, Roach, Gavin D., Shaw, Kit L., Terrazas, Dayna, White, Shanna L, Williams, David A.

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Phase 1 Study of CD37-Directed CAR T Cells in Patients with Relapsed or Refractory CD37+ Hematologic Malignancies von Frigault, Matthew J., Chen, Yi-Bin, Gallagher, Kathleen M.E., Horick, Nora K., El-Jawahri, Areej, Scarfò, Irene, Wehrli, Marc, Huang, Lu, Casey, Keagan, Cook, Daniella, Spitzer, Thomas, McAfee, Steven, Preffer, Frederic I., Armant, Myriam, Shaw, Kit L., Daley, Heather, Nikiforow, Sarah, Ritz, Jerome, Maus, Marcela V.

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Phase I Study of PHE885, a Fully Human BCMA-Directed CAR-T Cell Therapy for Relapsed/Refractory Multiple Myeloma Manufactured in <2 Days Using the T-Charge TM Platform von Sperling, Adam S., Nikiforow, Sarah, Nadeem, Omar, Mo, Clifton C, Laubach, Jacob P., Anderson, Kenneth C., Alonso, Alejandro, Ikegawa, Shuntaro, Prabhala, Rao, Hernandez Rodriguez, Diego, Daley, Heather, Shaw, Kit L., Arihara, Yohei, Ansari, Soudeh, Quinn, David S, Pearson, David, Hack, Anniesha, Treanor, Louise M, Bu, Dexiu, Mataraza, Jennifer, Rispoli, Lawrence, Credi, Marc, Ritz, Jerome, De Vita, Serena, Munshi, Nikhil C.

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240. Stable and Clinically Benign Clonal Dominance in an ADA-SCID Patient Treated With Retroviral Gene Therapy von Cooper, Aaron R., Shaw, Kit L., Lill, Georgia R., Sokolic, Robert, Candotti, Fabio, Kohn, Donald B.

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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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Characterization of the R572T Point Mutant of a Putative Cleavage Site in Human Foamy Virus Env von Bansal, A, Shaw, K L, Edwards, B H, Goepfert, P A, Mulligan, M J

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Abstract CT540: A phase 1 trial of cytokine-induced memory-like (CIML) natural killer (NK) cell therapy with IL-15 superagonist in advanced head and neck cancer: Part 1 results von Hanna, Glenn J., Coleman, Kimberly, Birch, Grace, Redd, Robert A., Alonso, Alejandro, Bednarz, Samantha, Daley, Heather, Hernandez Rodriguez, Diego E., Shaw, Kit L., Haddad, Robert I., Uppaluri, Ravindra, Ritz, Jerome, Nikiforow, Sarah, Soiffer, Robert J., Romee, Rizwan

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Autologous Transplant/Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immune Deficiency von Kohn, Donald B, Shaw, Kit L, Sokolic, Robert, MD, Carbonaro, Denise A, Davila, Alejandar, Barman, Provaboti, Garabedian, Elizabeth, Silvin, Christopher, De Oliveira, Satiro, MD, Shah, Ami J., MD, Moore, Theodore B., MD, Hershfield, Michael, Thrasher, Adrian, Gaspar, H. Robert, Candotti, Fabio

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30. Phase II Clinical Trial of Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA-SCID) Using a γ-Retroviral Vector von Shaw, Kit L., Garabedian, Elizabeth, Sokolic, Rob, Barman, Provaboti, Davila, Alejandra, Silvin, Christopher, de Oliveira, Satiro, Shah, Ami J., Terrazas, Dayna, Carbonaro, Denise, Geiger, Sabine, Mishra, Suparna, Cooper, Aaron, Smogorzewska, Monika, Jagadeesh, Jayashree, Hershfield, Michael S., Wayne, Alan, Crooks, Gay M., Moore, Theodore, Candotti, Fabio, Kohn, Donald B.

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Lentiviral vectors with amplified β cell-specific gene expression von Shaw, K L, Pais, E, Ge, S, Hardee, C, Skelton, D, Hollis, R P, Crooks, G M, Kohn, D B

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Busulfan Pharmacokinetics in ADA SCID Gene Therapy von Bradford, Kathryn L., Liu, Siyu, Krajinovic, Maja, Ansari, Marc, Garabedian, Elizabeth, Tse, John, Wang, Xiaoyan, Shaw, Kit L., Gaspar, H. Bobby, Candotti, Fabio, Kohn, Donald B.

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