Search Results - "Barese, Cecilia N."

Regulated Apoptosis of Genetically Modified Hematopoietic Stem and Progenitor Cells Via an Inducible Caspase‐9 Suicide Gene in Rhesus Macaques by Barese, Cecilia N., Felizardo, Tania C., Sellers, Stephanie E., Keyvanfar, Keyvan, Di Stasi, Antonio, Metzger, Mark E., Krouse, Allen E., Donahue, Robert E., Spencer, David M., Dunbar, Cynthia E.

“…The high risk of insertional oncogenesis reported in clinical trials using integrating retroviral vectors to genetically modify hematopoietic stem and…”
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Gene therapy for chronic granulomatous disease by Barese, Cecilia N, Goebel, W Scott, Dinauer, Mary C

“…Chronic granulomatous disease (CGD) is a congenital immune deficiency that is a promising therapeutic target for gene replacement into haematopoietic stem…”
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Screening chimeric GAA variants in preclinical study results in hematopoietic stem cell gene therapy candidate vectors for Pompe disease by Dogan, Yildirim, Barese, Cecilia N., Schindler, Jeffrey W., Yoon, John K., Unnisa, Zeenath, Guda, Swaroopa, Jacobs, Mary E., Oborski, Christine, Maiwald, Tim, Clarke, Diana L., Schambach, Axel, Pfeifer, Richard, Harper, Claudia, Mason, Chris, van Til, Niek P.

“…Pompe disease is a rare genetic neuromuscular disorder caused by acid α-glucosidase (GAA) deficiency resulting in lysosomal glycogen accumulation and…”
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Screening chimeric GAA variants in preclinicalstudy results in hematopoietic stem cell genetherapy candidate vectors for Pompe disease by Dogan, Yildirim, Barese, Cecilia N., Schindler, Jeffrey W., Yoon, John K., Unnisa, Zeenath, Guda, Swaroopa, Jacobs, Mary E., Oborski, Christine, Maiwald, Tim, Clarke, Diana L., Schambach, Axel, Pfeifer, Richard, Harper, Claudia, Mason, Chris, van Til, Niek P.

“…Pompe disease is a rare genetic neuromuscular disorder caused by acid α-glucosidase (GAA) deficiency resulting in lysosomal glycogen accumulation and…”
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Contributions of gene marking to cell and gene therapies by Barese, Cecilia N, Dunbar, Cynthia E

“…The first human genetic modification studies used replication-incompetent integrating vector vectors to introduce marker genes into T lymphocytes and…”
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Thymidine Kinase Suicide Gene-mediated Ganciclovir Ablation of Autologous Gene-modified Rhesus Hematopoiesis by Barese, Cecilia N, Krouse, Allen E, Metzger, Mark E, King, Connor A, Traversari, Catia, Marini, Frank C, Donahue, Robert E, Dunbar, Cynthia E

“…Despite the genotoxic complications encountered in clinical gene therapy trials for primary immunodeficiency diseases targeting hematopoietic cells with…”
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Recurrent eosinophilic cystitis in a child with chronic granulomatous disease by Barese, Cecilia N, Podestá, Miguel, Litvak, Edith, Villa, Mariana, Rivas, Eva María

“…Eosinophilic cystitis is an uncommon disease in children, and its association with chronic granulomatous disease (CGD) has been previously reported in only…”
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Rac2 concentrations in umbilical cord neutrophils by Meade, Virginia M, Barese, Cecilia N, Kim, Chaekyun, Njinimbam, Charles G, Marchal, Christophe C, Ingram, David A, Clapp, D Wade, Dinauer, Mary C, Yoder, Mervin C

“…Human newborn infants display a variety of immunodeficiencies of immaturity, including diminished neutrophil adhesion, chemotaxis, and migration. Rac2, a…”
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Molecular characterization of a novel splice site mutation within the CYBB gene leading to X-linked chronic granulomatous disease by Barese, Cecilia N., Copelli, Silvia B., Matteo, Elena De, Zandomeni, Rubén, Salgueiro, Fabián, Giovanni, Daniela Di, Heyworth, Paul, Rivas, Eva María

“…Chronic granulomatous disease (CGD) is a primary immunodeficiency that affects the oxidative mechanism of microbial killing of phagocytic cells. The defect is…”
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Thymidine Kinase Suicide Gene-mediated Ganciclovir Ablation of Autologous Gene-modified Rhesus Hematopoiesis by Barese, Cecilia N, Krouse, Allen E, Metzger, Mark E, King, Connor A, Traversari, Catia, Marini, Frank C, Donahue, Robert E, Dunbar, Cynthia E

“…Despite the genotoxic complications encountered in clinical gene therapy trials for primary immunodeficiency diseases targeting hematopoietic cells with…”
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Retroviral vector integration in post-transplant hematopoiesis in mice conditioned with either submyeloablative or ablative irradiation by Sadat, M A, Dirscherl, S, Sastry, L, Dantzer, J, Pech, N, Griffin, S, Hawkins, T, Zhao, Y, Barese, C N, Cross, S, Orazi, A, An, C, Goebel, W S, Yoder, M C, Li, X, Grez, M, Cornetta, K, Mooney, S D, Dinauer, M C

“…X-linked chronic granulomatous disease (X-CGD) is an inherited immunodeficiency with absent phagocyte NADPH-oxidase activity caused by defects in the…”
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Granulocyte Colony-Stimulating Factor Prior to Low-Dose Total Body Irradiation as Conditioning Regimen in Hematopoietic Stem Cell Transplantation for Gene Therapy by Barese, Cecilia N., Goebel, W. Scott, Pech, Nancy K., Meyers, Justin, Dinauer, Mary C.

“…Genetic blood diseases can potentially be treated by transplantation of autologous hematopoietic stem cells (HSC) transduced with the functional gene…”
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