المؤلفون: 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

المصدر: New England Journal of Medicine. 384(21)

مصطلحات موضوعية: Medical Biotechnology, Biomedical and Clinical Sciences, Biotechnology, Transplantation, Gene Therapy, Genetics, Regenerative Medicine, Clinical Research, Stem Cell Research, Stem Cell Research - Nonembryonic - Human, Evaluation of treatments and therapeutic interventions, 5.2 Cellular and gene therapies, Development of treatments and therapeutic interventions, 6.2 Cellular and gene therapies, Good Health and Well Being, Adenosine Deaminase, Adolescent, Agammaglobulinemia, Child, Child, Preschool, Genetic Therapy, Genetic Vectors, Hematopoietic Stem Cell Transplantation, Humans, Infant, Lentivirus, Lymphocyte Count, Progression-Free Survival, Prospective Studies, Severe Combined Immunodeficiency, Transplantation, Autologous, Medical and Health Sciences, General & Internal Medicine, Biomedical and clinical sciences, Health sciences

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/0tw1k4mm

المؤلفون: Hammond, Terese C.^{Aff1, Aff2}, Purbhoo, Marco A., Kadel, Sapana, Ritz, Jerome, Nikiforow, Sarah, Daley, Heather, Shaw, Kit, van Besien, Koen, Gomez-Arteaga, Alexandra, Stevens, Don, Ortuzar, Waldo, Michelet, Xavier, Smith, Rachel, Moskowitz, Darrian, Masakayan, Reed, Yigit, Burcu, Boi, Shannon, Soh, Kah Teong, Chamberland, John^{Aff3, Aff8}, Song, Xin, Qin, Yu^{Aff3, Aff8}, Mishchenko, Ilya, Kirby, Maurice, Nasonenko, Valeriia, Buffa, Alexa^{Aff3, Aff8}, Buell, Jennifer S., Chand, Dhan, van Dijk, Marc, Stebbing, Justin^{Aff9, IDs4146702444905z_cor29}, Exley, Mark A.

المصدر: Nature Communications. 15(1)

المؤلفون: 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

المصدر: Transplantation and Cellular Therapy. 26(10)

مصطلحات موضوعية: Medical Biotechnology, Pharmacology and Pharmaceutical Sciences, Biomedical and Clinical Sciences, Stem Cell Research - Nonembryonic - Human, Stem Cell Research, Clinical Research, Gene Therapy, Genetics, Regenerative Medicine, Stem Cell Research - Nonembryonic - Non-Human, Biotechnology, Clinical Trials and Supportive Activities, Transplantation, Adenosine Deaminase, Agammaglobulinemia, Busulfan, Child, Genetic Therapy, Hematopoietic Stem Cell Transplantation, Humans, Infant, Severe Combined Immunodeficiency, Transplantation Conditioning, Adenosine deaminase, SCID, Pharmacokinetics, Gene therapy, Clinical trials, Clinical Sciences, Immunology, Cardiovascular medicine and haematology

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/7wh2h4v0

المؤلفون: 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, Net4CGD consortium

المصدر: Nature medicine. 26(2)

مصطلحات موضوعية: Net4CGD consortium, Neutrophils, Hematopoietic Stem Cells, Chromosomes, Human, X, Humans, Lentivirus, Granulomatous Disease, Chronic, Antigens, CD34, Treatment Outcome, Transplantation Conditioning, Comorbidity, Gene Silencing, Genes, Regulator, Genetic Vectors, Adolescent, Child, Child, Preschool, United States, Male, Promoter Regions, Genetic, Young Adult, Patient Safety, Genetic Therapy, United Kingdom, NADPH Oxidases, Genetics, Hematology, Regenerative Medicine, Stem Cell Research, Stem Cell Research - Nonembryonic - Human, Gene Therapy, Clinical Trials and Supportive Activities, Infectious Diseases, Clinical Research, 5.2 Cellular and gene therapies, Development of treatments and therapeutic interventions, Inflammatory and immune system, Infection, Medical and Health Sciences, Immunology

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/7b61w03m

المؤلفون: Frigault, Matthew J., Bishop, Michael R., Rosenblatt, Jacalyn, O’Donnell, Elizabeth K., Raje, Noopur, Cook, Daniella, Yee, Andrew J., Logan, Emma, Avigan, David E., Jakubowiak, Andrzej, Shaw, Kit, Daley, Heather, Nikiforow, Sarah, Griffin, Faith, Cornwell, Christine, Shen, Angela, Heery, Christopher, Maus, Marcela V.

المصدر: In Blood Advances April 2022

المؤلفون: 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. ^*

المصدر: In Blood 14 October 2021 138(15):1304-1316

المؤلفون: 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.

المصدر: In Biology of Blood and Marrow Transplantation October 2020 26(10):1819-1827

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المؤلفون: Cooper, Aaron R., Lill, Georgia R., Shaw, Kit, Carbonaro-Sarracino, Denise A., Davila, Alejandra, Sokolic, Robert, Candotti, Fabio, Pellegrini, Matteo, Kohn, Donald B.

المصدر: In Blood 11 May 2017 129(19):2624-2635

المؤلفون: 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.

المصدر: In Blood 15 November 2022 140 Supplement 1:10665-10667