Cytoreductive conditioning intensity predicts clonal diversity in ADA-SCID retroviral gene therapy patients
Cooper, Aaron R., Lill, Georgia R., Shaw, Kit, Carbonaro-Sarracino, Denise A., Davila, Alejandra, Sokolic, Robert, Candotti, Fabio, Pellegrini, Matteo, Kohn, Donald B.
Published in Blood (11.05.2017)
Published in Blood (11.05.2017)
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Journal Article
Gene delivery using AAV8 in vivo for disease stabilization in a bimodal gene therapy approach for the treatment of ADA-deficient SCID
Carbonaro-Sarracino, Denise A., Chun, Krista, Clark, Danielle N., Kaufman, Michael L., Jin, Xiangyang, Wang, Xiaoyan, Kohn, Donald B.
Published in Molecular therapy. Methods & clinical development (12.03.2021)
Published in Molecular therapy. Methods & clinical development (12.03.2021)
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Journal Article
Dosing and Re-Administration of Lentiviral Vector for In Vivo Gene Therapy in Rhesus Monkeys and ADA-Deficient Mice
Carbonaro-Sarracino, Denise A., Tarantal, Alice F., Lee, C. Chang I., Kaufman, Michael L., Wandro, Stephen, Jin, Xiangyang, Martinez, Michele, Clark, Danielle N., Chun, Krista, Koziol, Colin, Hardee, Cinnamon L., Wang, Xiaoyan, Kohn, Donald B.
Published in Molecular therapy. Methods & clinical development (13.03.2020)
Published in Molecular therapy. Methods & clinical development (13.03.2020)
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Journal Article
Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID): Molecular Pathogenesis and Clinical Manifestations
Bradford, Kathryn L., Moretti, Federico A., Carbonaro-Sarracino, Denise A., Gaspar, Hubert B., Kohn, Donald B.
Published in Journal of clinical immunology (01.10.2017)
Published in Journal of clinical immunology (01.10.2017)
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Journal Article
Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency
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.
Published in Blood (14.10.2021)
Published in Blood (14.10.2021)
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Journal Article
Effects of Vector Backbone and Pseudotype on Lentiviral Vector-mediated Gene Transfer: Studies in Infant ADA-Deficient Mice and Rhesus Monkeys
Carbonaro Sarracino, Denise, Tarantal, Alice F, Lee, C Chang I., Martinez, Michele, Jin, Xiangyang, Wang, Xiaoyan, Hardee, Cinnamon L, Geiger, Sabine, Kahl, Christoph A, Kohn, Donald B
Published in Molecular therapy (01.10.2014)
Published in Molecular therapy (01.10.2014)
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Journal Article
Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency
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
Published in The New England journal of medicine (27.05.2021)
Published in The New England journal of medicine (27.05.2021)
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Journal Article
Letter to the Editor. Differences between lentiviral vector-mediated ex vivo hematopoietic stem cell and adeno-associated virus gene therapies for neurological disorders
Carbonaro-Sarracino, Denise A, LeWinter, Robin, Meltzer, Leslie
Published in Journal of neurosurgery (10.12.2021)
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Published in Journal of neurosurgery (10.12.2021)
Journal Article
442. Decreased Survival and Engraftment in Adult ADA-Deficient Mice Co-Transplanted With Uncorrected Lineage-Positive Bone Marrow Cells Combined With Gene-Corrected Lineage-Negative Bone Marrow Cells
Carbonaro-Sarracino, Denise A., Kaufman, Michael L., Jin, Xiangyang, Geiger, Sabine, Clark, Danielle, Kohn, Donald B.
Published in Molecular therapy (01.05.2015)
Published in Molecular therapy (01.05.2015)
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Journal Article
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
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
Published in Blood (13.11.2019)
Published in Blood (13.11.2019)
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Journal Article
764. Dosing and Re-Administration of Intravenous Lentiviral Vector for Liver-Directed Gene Transfer in Young Rhesus Monkeys and ADA-Deficient Mice
Carbonaro-Sarracino, Denise A., Tarantal, Alice F., Lee, Chang I., Kaufman, Michael L., Martinez, Michele, Clark, Danielle N., Koziol, Colin, Kohn, Donald B.
Published in Molecular therapy (01.05.2016)
Published in Molecular therapy (01.05.2016)
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Journal Article
Gene therapy/bone marrow transplantation in ADA-deficient mice: roles of enzyme-replacement therapy and cytoreduction
Carbonaro, Denise A., Jin, Xiangyang, Wang, Xingchao, Yu, Xiao-Jin, Rozengurt, Nora, Kaufman, Michael L., Wang, Xiaoyan, Gjertson, David, Zhou, Yang, Blackburn, Michael R., Kohn, Donald B.
Published in Blood (01.11.2012)
Published in Blood (01.11.2012)
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