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Your search keyword '"Smogorzewska, E. Monika"' showing total 7 results
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7 results on '"Smogorzewska, E. Monika"'

2. Clinical efficacy of gene-modified stem cells in adenosine deaminase–deficient immunodeficiency

Author

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

Published
2017
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3. Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report

Author

Engel, Barbara C., primary, Podsakoff, Greg M., additional, Ireland, Joanna L., additional, Smogorzewska, E. Monika, additional, Carbonaro, Denise A., additional, Wilson, Kathy, additional, Shah, Ami, additional, Kapoor, Neena, additional, Sweeney, Mirna, additional, Borchert, Mark, additional, Crooks, Gay M., additional, Weinberg, Kenneth I., additional, Parkman, Robertson, additional, Rosenblatt, Howard M., additional, Wu, Shi-Qi, additional, Hershfield, Michael S., additional, Candotti, Fabio, additional, and Kohn, Donald B., additional

Published
2006
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4. T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates

Author

Kohn, Donald B., primary, Hershfield, Michal S., additional, Carbonaro, Denise, additional, Shigeoka, Ann, additional, Brooks, Judith, additional, Smogorzewska, E. Monika, additional, Barsky, Lora W., additional, Chan, Raymond, additional, Burotto, Felix, additional, Annett, Geralyn, additional, Nolta, Jan A., additional, Crooks, Gay, additional, Kapoor, Neena, additional, Eldetr, Melissa, additional, Wara, Diane, additional, Bowen, Thomas, additional, Madsen, Edward, additional, Synder, Floyd F., additional, Bastian, John, additional, Muul, Linda, additional, Blaese, R. Michael, additional, Weinberg, Kenneth, additional, and Parkman, Robertson, additional

Published
1998
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5. T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates.

Author

Kohn, Donald B., Hershfield, Michal S., Carbonaro, Denise, Shigeoka, Ann, Brooks, Judith, Smogorzewska, E. Monika, Barsky, Lora W., Chan, Raymond, Burotto, Felix, Annett, Geralyn, Nolta, Jan A., Crooks, Gay, Kapoor, Neena, Eldetr, Melissa, Wara, Diane, Bowen, Thomas, Madsen, Edward, Synder, Floyd F., Bastian, John, and Muul, Linda

Published
1998
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6. Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report

Author

Engel, Barbara C., Podsakoff, Greg M., Ireland, Joanna L., Smogorzewska, E. Monika, Carbonaro, Denise A., Wilson, Kathy, Shah, Ami, Kapoor, Neena, Sweeney, Mirna, Borchert, Mark, Crooks, Gay M., Weinberg, Kenneth I., Parkman, Robertson, Rosenblatt, Howard M., Wu, Shi-Qi, Hershfield, Michael S., Candotti, Fabio, and Kohn, Donald B.

Abstract

A patient with adenosine deaminase–deficient severe combined immune deficiency (ADA-SCID) was enrolled in a study of retroviral-mediated ADA gene transfer to bone marrow hematopoietic stem cells. After the discontinuation of ADA enzyme replacement, busulfan (75 mg/m2) was administered for bone marrow cytoreduction, followed by infusion of autologous, gene-modified CD34+ cells. The expected myelosuppression developed after busulfan but then persisted, necessitating the administration of untransduced autologous bone marrow back-up at day 40. Because of sustained pancytopenia and negligible gene marking, diagnostic bone marrow biopsy and aspirate were performed at day 88. Analyses revealed hypocellular marrow and, unexpectedly, evidence of trisomy 8 in 21.6% of cells. Trisomy 8 mosaicism (T8M) was subsequently diagnosed by retrospective analysis of a pretreatment marrow sample that might have caused the lack of hematopoietic reconstitution. The confounding effects of this preexisting marrow cytogenetic abnormality on the response to gene transfer highlights another challenge of gene therapy with the use of autologous hematopoietic stem cells.

Published
2007
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7. T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+cells in ADA-deficient SCID neonates

Author

Kohn, Donald B., Hershfield, Michal S., Carbonaro, Denise, Shigeoka, Ann, Brooks, Judith, Smogorzewska, E. Monika, Barsky, Lora W., Chan, Raymond, Burotto, Felix, Annett, Geralyn, Nolta, Jan A., Crooks, Gay, Kapoor, Neena, Eldetr, Melissa, Wara, Diane, Bowen, Thomas, Madsen, Edward, Synder, Floyd F., Bastian, John, Muul, Linda, Blaese, R. Michael, Weinberg, Kenneth, and Parkman, Robertson

Abstract

Adenosine deaminase-deficient severe combined immunodeficiency was the first disease investigated for gene therapy because of a postulated production or survival advantage for genecorrected T lymphocytes, which may overcome inefficient gene transfer. Four years after three newborns with this disease were given infusions of transduced autologous umbilical cord blood CD34+cells, the frequency of gene-containing T lymphocytes has risen to 1–10%, whereas the frequencies of other hematopoietic and lymphoid cells containing the gene remain at 0.01–0.1%. Cessation of polyethylene glycol-conjugated adenosine deaminase enzyme replacement in one subject led to a decline in immune function, despite the persistence of gene-containing T lymphocytes. Thus, despite the long-term engraftment of transduced stem cells and selective accumulation of gene-containing T lymphocytes, improved gene transfer and expression will be needed to attain a therapeutic effect.

Published
1998
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