Search Results - "Shupien, Sally"

Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency by 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

“…Fifty patients in the United States and the United Kingdom underwent HSCT with CD34+ cells transduced with a replication-defective lentivirus bearing ADA . At…”
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Clinical efficacy of gene-modified stem cells in adenosine deaminase-deficient immunodeficiency by 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

“…Autologous hematopoietic stem cell transplantation (HSCT) of gene-modified cells is an alternative to enzyme replacement therapy (ERT) and allogeneic HSCT that…”
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Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency by 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.

“…Patients lacking functional adenosine deaminase activity have severe combined immunodeficiency (ADA SCID), which can be treated with ADA enzyme replacement…”
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β-globin gene transfer to human bone marrow for sickle cell disease by Romero, Zulema, Urbinati, Fabrizia, Geiger, Sabine, Cooper, Aaron R, Wherley, Jennifer, Kaufman, Michael L, Hollis, Roger P, de Assin, Rafael Ruiz, Senadheera, Shantha, Sahagian, Arineh, Jin, Xiangyang, Gellis, Alyse, Wang, Xiaoyan, Gjertson, David, Deoliveira, Satiro, Kempert, Pamela, Shupien, Sally, Abdel-Azim, Hisham, Walters, Mark C, Meiselman, Herbert J, Wenby, Rosalinda B, Gruber, Theresa, Marder, Victor, Coates, Thomas D, Kohn, Donald B

“…Autologous hematopoietic stem cell gene therapy is an approach to treating sickle cell disease (SCD) patients that may result in lower morbidity than…”
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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 (ADA-SCID): Results in an Expanded Cohort by 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

“…Background: Severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID) is a rare disorder caused by ADA gene mutations, leading to…”
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[Beta]-globin gene transfer to human bone marrow for sickle cell disease by Romero, Zulema, Urbinati, Fabrizia, Geiger, Sabine, Cooper, Aaron R, Wherley, Jennifer, Kaufman, Michael L, Hollis, Roger P, de Assin, Rafael Ruiz, Senadheera, Shantha, Sahagian, Arineh, Jin, Xiangyang, Gellis, Alyse, Wang, Xiaoyan, Gjertson, David, DeOliveira, Satiro, Kempert, Pamela, Shupien, Sally, Abdel-Azim, Hisham, Walters, Mark C, Meiselman, Herbert J, Wenby, Rosalinda B, Gruber, Theresa, Marder, Victor, Coates, Thomas D, Kohn, Donald B

“…Autologous hematopoietic stem cell gene therapy is an approach to treating sickle cell disease (SCD) patients that may result in lower morbidity than…”
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Somatic Gene Therapy for X-Linked Severe Combined Immunodeficiency Using a Self-Inactivating Modified Gammaretroviral Vector Results in An Improved Preclinical Safety Profile and Early Clinical Efficacy in a Human Patient by Pai, Sung-Yun, Notarangelo, Luigi Daniele, Harris, Chad, Cattaneo, Federica, Wladkowski, Matthew, Armant, Myriam, Kao, Grace S., Wielgosz, Matthew, Nienhuis, Arthur W., Modlich, Ute, Brugman, Martijn, Schambach, Axel, Rothe, Michael, Thornhill, Susannah I, Gaspar, H. Bobby, Parsley, Kathryn, Cooray, Samantha, Grassman, Elke, Bushman, Frederic, Shupien, Sally, Oleastro, Matias, Hacein-Bey-Abina, Salima, Fischer, Alain, Cavazzana-Calvo, Marina, Malik, Punam, Filipovich, Alexandra, Kohn, Donald B., Baum, Christopher, Williams, David A., Thrasher, Adrian J

“…Abstract 164▪FN2▪This icon denotes a clinically relevant abstract Somatic gene therapy for X-linked severe combined immunodeficiency (X-SCID) using a MLV-based…”
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Metformin extended release treatment of adolescent obesity: a 48-week randomized, double-blind, placebo-controlled trial with 48-week follow-up by Wilson, Darrell M, Abrams, Stephanie H, Aye, Tandy, Lee, Phillip D K, Lenders, Carine, Lustig, Robert H, Osganian, Stavroula V, Feldman, Henry A

“…Metformin has been proffered as a therapy for adolescent obesity, although long-term controlled studies have not been reported. To test the hypothesis that 48…”
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