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Journal Articles

Enabling Gene-Edited, Regulatory-like, T Cells (edTreg) for Treatment of IPEX and Other Autoimmune Disorders

Yuchi Honaker, PhD, Karen Sommer, PhD, Noelle Dahl, Yufei Xiang, MD, PhD, Christina Lopez, PhD, Samuel Scharffenberger, Michelle Christian, Troy R. Torgerson, MD PhD, Andrew M. Scharenberg, MD, David J. Rawlings, MD

Blood (2019) 134 (Supplement_1): 2071.

https://doi.org/10.1182/blood-2019-131946

Published: 2019

Journal Articles

Conversion of T-Effector Cells to Immunosuppressive T-Regulatory-like Cells By CRISPR/Cas9-Mediated Integration of a FOXP3 Transgene

Yuchi Honaker, PhD, Yufei Xiang, MD, PhD, Logan Fisher, B.S., Karen Sommer, PhD, Troy R. Torgerson, MD PhD, Andrew M. Scharenberg, MD, David J. Rawlings, MD

Blood (2018) 132 (Supplement 1): 3490.

https://doi.org/10.1182/blood-2018-99-117456

Published: 2018

Journal Articles

Genetic Disorders of Immune Tolerance: The Flip Side of Immune Deficiency Where Autoimmunity Trumps Infection

Troy R. Torgerson, MDPhD

Blood (2017) 130 (Supplement 1): SCI-29.

https://doi.org/10.1182/blood.V130.Suppl_1.SCI-29.SCI-29

Published: 2017

Journal Articles

Development of a Treg Lineage Specific Lentiviral Vector for the Treatment of IPEX Syndrome

Katelyn E Masiuk, BS, Jennifer Laborada, BS, Roger P Hollis, PhD, Donald B Kohn, MD

Blood (2017) 130 (Supplement 1): 528.

https://doi.org/10.1182/blood.V130.Suppl_1.528.528

Published: 2017

Journal Articles

Generation of Functional Regulatory T Cells By FOXP3 Gene Transfer into CD4 T Cells from Scurfy Mice and IPEX Patients

Marianne Delville, MD, Emmanuelle Six, PhD, Florence Bellier, Nelly Sigrist, David Zemmour, PhD, Marie-André Bessard, MD, Fabienne Mazerolles, PhD, David Gross, PhD, Axel Schambach, MD PhD, Frederic Rieux-Laucat, PhD, Christoph Benoist, MD PhD, Isabelle Andre-Schmutz, Marina Cavazzana, MD PhD

Blood (2016) 128 (22): 2526.

https://doi.org/10.1182/blood.V128.22.2526.2526

Published: 2016

Journal Articles

Outcomes after Donor Lymphocyte Infusion for Insufficient Donor Chimerism Following Hematopoietic Cell Transplantation for Non-Malignant Disorders

Carter Biewen, BA, Angela R Smith, MD MS, Jakub Tolar, MD, PhD, Weston P Miller, IV, MD

Blood (2015) 126 (23): 1969.

https://doi.org/10.1182/blood.V126.23.1969.1969

Published: 2015

Journal Articles

Immunological Derangement in Follicular Lymphoma Patients Developing Severe Side Effects during Treatment with PI3K Inhibitors

Marina Motta, MD, Marco Chiarini, MSC, Marta Petullà, PharmD, Claudia Ghidini, BSC, Marion Vaglio Tessitore, BSC, Viviana Giustini, MSC, Chiara Pagani, MD, Antonella Anastasia, MD, Samantha Ferrari, MD, Alessandra Tucci, Luisa Imberti, MD, Giuseppe Rossi, MD

Blood (2015) 126 (23): 1531.

https://doi.org/10.1182/blood.V126.23.1531.1531

Published: 2015

Journal Articles

Fatal autoimmunity in mice reconstituted with human hematopoietic stem cells encoding defective FOXP3

Blood (2015) 125 (25): 3886–3895.

https://doi.org/10.1182/blood-2014-12-618363

Published: 2015

Includes: Supplemental data

Journal Articles

Robust Immune Reconstitution in Children with Severe Primary Immunodeficiency after Reduced-Intensity Conditioning Hematopoietic Stem Cell Transplantation

William T. Tse, MD PhD, Jessica Ward, MSN, cPNP, Jennifer Schneiderman, MD MS, Sonali Chaudhury, MD, Ramsay Fuleihan, MD, Reggie Duerst, MD, Morris Kletzel, MD

Blood (2014) 124 (21): 3923.

https://doi.org/10.1182/blood.V124.21.3923.3923

Published: 2014

Journal Articles

T-Cell-Mediated Suppression: From Bench to Bedside

Maria Grazia Roncarolo, MD, Manuela Battaglia, PhD, Rosa Bacchetta, MD, Megan Levings, PhD, Silvia Gregori, PhD

Blood (2013) 122 (21): SCI-37.

https://doi.org/10.1182/blood.V122.21.SCI-37.SCI-37

Published: 2013

Journal Articles

Accumulation of peripheral autoreactive B cells in the absence of functional human regulatory T cells

Tuure Kinnunen, Nicolas Chamberlain, Henner Morbach, Jinyoung Choi, Sangtaek Kim, Joseph Craft, Lloyd Mayer, Caterina Cancrini, Laura Passerini, Rosa Bacchetta, Hans D. Ochs, Troy R. Torgerson, Eric Meffre

Blood (2013) 121 (9): 1595–1603.

https://doi.org/10.1182/blood-2012-09-457465

Published: 2013

Includes: Supplemental data

Journal Articles

HLA Haploidentical Stem Cell Transplantation After Removal of αβ + T Lymphocytes and B Lymphocytes Is an Effective Treatment for Children with Life-Threatening, Non-Malignant Disorders

Alice Bertaina, MD, Mariateresa Romano, MD, Sergio Rutella, MD PhD, Giuseppe Palumbo, MD, Daria Pagliara, MD, Stefano Ceccarelli, MD, Letizia P Brescia, MD, Giuseppina Li Pira, BSc, Francesca Cocca, MD, Maria Ester Bernardo, MD, Giuseppe M Milano, MD, Daniela Pende, PhD, Michela Falco, BSc, Lorenzo Moretta, MD, Franco Locatelli, MD, PhD

Blood (2012) 120 (21): 2018.

https://doi.org/10.1182/blood.V120.21.2018.2018

Published: 2012

Journal Articles

Selective engraftment of donor CD4 + 25 high FOXP3-positive T cells in IPEX syndrome after nonmyeloablative hematopoietic stem cell transplantation

Markus G. Seidel, Gerhard Fritsch, Thomas Lion, Birgit Jürgens, Andreas Heitger, Rosa Bacchetta, Anita Lawitschka, Christina Peters, Helmut Gadner, Susanne Matthes-Martin

Blood (2009) 113 (22): 5689–5691.

https://doi.org/10.1182/blood-2009-02-206359

Published: 2009

Journal Articles

Potential Increase in Graft Failure Risk after Reduced Intensity Conditioning Stem Cell Transplant (RIT) Using Umbilical Cord Blood for Children with Primary Immunodeficiency.

Jessica Guarino, MSN, MPH, Morris Kletzel, M.D., Reggie Duerst, M.D., David Jacobsohn, M.D., M.S., Jennifer Schneiderman, M.D., M.S., William T. Tse, M.D., Ph.D.

Blood (2007) 110 (11): 2006.

https://doi.org/10.1182/blood.V110.11.2006.2006

Published: 2007

Journal Articles

Submyeloablative Cord Blood Transplant Corrects Clinical Defects Seen in IPEX Syndrome.

Kenneth G. Lucas, MD, Melanie A. Comito, MD, David R. Ungar, MD, Brandt P. Groh, MD

Blood (2006) 108 (11): 5414.

https://doi.org/10.1182/blood.V108.11.5414.5414

Published: 2006

Journal Articles

Successful bone marrow transplantation for IPEX syndrome after reduced-intensity conditioning

Aarati Rao, Naynesh Kamani, Alexandra Filipovich, Susan Molleran Lee, Stella M. Davies, Jignesh Dalal, Shalini Shenoy

Blood (2006) 109 (1): 383–385.

https://doi.org/10.1182/blood-2006-05-025072

Published: 2006

Journal Articles

Postgrafting Immune Suppression Combined with Nonmyeloablative Conditioning for Transplantation of HLA-Identical Hematopoietic Cell Grafts: Results of a Phase I Study for Treatment of Immunodeficiency Disorders.

Lauri Burroughs, MD, Rainer F. Storb, MD, Wendy Leisenring, Michael Pulsipher, MD, Michael R. Loken, PhD, Troy Torgerson, MD, Hans D. Ochs, MD, Ann Woolfrey, MD

Blood (2005) 106 (11): 327.

https://doi.org/10.1182/blood.V106.11.327.327

Published: 2005

Journal Articles

FOXP3 Expression in B and T Cell Lymphomas.

Giovanna Roncador, Juan Fernando Garcia, Jose Francisco Garcia, Lorena Maestre, Elena Lucas, Juan Cruz Cigudosa, Lydia Sanchez, Koichi Ohshima, Shigeo Nakamura, Alison Banham, Miguel Angel Piris

Blood (2005) 106 (11): 4503.

https://doi.org/10.1182/blood.V106.11.4503.4503

Published: 2005

Journal Articles

Reduced Intensity Conditioning Therapy Using Campath -1H Is Successful for Stem Cell Transplantation in Non-Malignant Disorders.

Aarati Rao, MD, Robert Hayashi, MD, William Grossman, MD, PhD, David Wilson, MD, PhD, Lolie Yu, MD, John DiPersio, MD, PhD, Yvonne Barnes, PNP, Shalini Shenoy, MD

Blood (2004) 104 (11): 1823.

https://doi.org/10.1182/blood.V104.11.1823.1823

Published: 2004

NARROW

Enabling Gene-Edited, Regulatory-like, T Cells (edTreg) for Treatment of IPEX and Other Autoimmune Disorders

Conversion of T-Effector Cells to Immunosuppressive T-Regulatory-like Cells By CRISPR/Cas9-Mediated Integration of a FOXP3 Transgene

Genetic Disorders of Immune Tolerance: The Flip Side of Immune Deficiency Where Autoimmunity Trumps Infection

Development of a Treg Lineage Specific Lentiviral Vector for the Treatment of IPEX Syndrome

Generation of Functional Regulatory T Cells By FOXP3 Gene Transfer into CD4 T Cells from Scurfy Mice and IPEX Patients

Outcomes after Donor Lymphocyte Infusion for Insufficient Donor Chimerism Following Hematopoietic Cell Transplantation for Non-Malignant Disorders

Immunological Derangement in Follicular Lymphoma Patients Developing Severe Side Effects during Treatment with PI3K Inhibitors

Fatal autoimmunity in mice reconstituted with human hematopoietic stem cells encoding defective FOXP3

Robust Immune Reconstitution in Children with Severe Primary Immunodeficiency after Reduced-Intensity Conditioning Hematopoietic Stem Cell Transplantation

T-Cell-Mediated Suppression: From Bench to Bedside

Accumulation of peripheral autoreactive B cells in the absence of functional human regulatory T cells

HLA Haploidentical Stem Cell Transplantation After Removal of αβ + T Lymphocytes and B Lymphocytes Is an Effective Treatment for Children with Life-Threatening, Non-Malignant Disorders

Selective engraftment of donor CD4 + 25 high FOXP3-positive T cells in IPEX syndrome after nonmyeloablative hematopoietic stem cell transplantation

Potential Increase in Graft Failure Risk after Reduced Intensity Conditioning Stem Cell Transplant (RIT) Using Umbilical Cord Blood for Children with Primary Immunodeficiency.

Submyeloablative Cord Blood Transplant Corrects Clinical Defects Seen in IPEX Syndrome.

Successful bone marrow transplantation for IPEX syndrome after reduced-intensity conditioning

Postgrafting Immune Suppression Combined with Nonmyeloablative Conditioning for Transplantation of HLA-Identical Hematopoietic Cell Grafts: Results of a Phase I Study for Treatment of Immunodeficiency Disorders.

FOXP3 Expression in B and T Cell Lymphomas.

Reduced Intensity Conditioning Therapy Using Campath -1H Is Successful for Stem Cell Transplantation in Non-Malignant Disorders.

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American Society of Hematology

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American Society of Hematology

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