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Journal Articles

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

Donald B. Kohn, MD, Kit L. Shaw, PhD, Elizabeth Garabedian, RN, MS, Denise Ann Carbonaro-Sarracino, PhD, Theodore B. Moore, MD, Satiro N. De Oliveira, MD, Gay M. Crooks, MD, John Tse, Sally Shupien, Dayna Terrazas, Alejandra Davila, Amalia Icreverzi, PhD, Allen Yu, Krista M. Chun, Christian E. Casas, Provaboti Barman, PhD, Maritess Coronel, Beatriz Campo Fernandez, PhD, Ruixue Zhang, Roger P. Hollis, PhD, Chilenwa Uzowuru, Hilory Ricketts, Jinhua Xu Bayford, Valentina Trevisan, Serena Arduini, PhD, Frances Lynn, Mahesh Kudari, MBBS, MA, Andrea Spezzi, MD MBBS, Lilith Reeves, MS, MT(ASCP), Kenneth Cornetta, Robert A. Sokolic, MD, Roberta Parrott, BS, Rebecca Buckley, MD, Claire Booth, Fabio Candotti, MD, Harry L. Malech, MD, Adrian J. Thrasher, MBBS, PhD FMedSci, H. Bobby Gaspar, MBBS, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3345.
https://doi.org/10.1182/blood-2019-123432
Published: 2019
Journal Articles

Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency

Maria Pia Cicalese, Francesca Ferrua, Laura Castagnaro, Roberta Pajno, Federica Barzaghi, Stefania Giannelli, Francesca Dionisio, Immacolata Brigida, Marco Bonopane, Miriam Casiraghi, Antonella Tabucchi, Filippo Carlucci, Eyal Grunebaum, Mehdi Adeli, Robbert G. Bredius, Jennifer M. Puck, Polina Stepensky, Ilhan Tezcan, Katie Rolfe, Erika De Boever, Rickey R. Reinhardt, Jonathan Appleby, Fabio Ciceri, Maria Grazia Roncarolo, Alessandro Aiuti
Journal: Blood
Blood (2016) 128 (1): 45–54.
https://doi.org/10.1182/blood-2016-01-688226
Published: 2016
Includes: Supplemental data
Journal Articles

Adenosine Signaling in Priapism and Novel Therapies

Chen Ning, Jiaming Wen, Yujin Zhang, Harinder S. Juneja, Lin Qi, Rodney E. Kellems, Yang Xia
Journal: Blood
Blood (2012) 120 (21): 1006.
https://doi.org/10.1182/blood.V120.21.1006.1006
Published: 2012
Journal Articles

Alterations in the adenosine metabolism and CD39/CD73 adenosinergic machinery cause loss of Treg cell function and autoimmunity in ADA-deficient SCID

Aisha V. Sauer, Immacolata Brigida, Nicola Carriglio, Raisa Jofra Hernandez, Samantha Scaramuzza, Daniela Clavenna, Francesca Sanvito, Pietro L. Poliani, Nicola Gagliani, Filippo Carlucci, Antonella Tabucchi, Maria Grazia Roncarolo, Elisabetta Traggiai, Anna Villa, Alessandro Aiuti
Journal: Blood
Blood (2012) 119 (6): 1428–1439.
https://doi.org/10.1182/blood-2011-07-366781
Published: 2012
Includes: Supplemental data
Journal Articles

Correction of Immune and Enzyme Deficiency by Bone Marrow Transplant in PNP Deficient SCID

Vikramajit Singh, MRCP, Robert Wynn, MD, Denise Bonney, PhD, Stephen Hughes, MD, Kay Poulton, PhD, Helena Lee, PhD
Journal: Blood
Blood (2011) 118 (21): 4556.
https://doi.org/10.1182/blood.V118.21.4556.4556
Published: 2011
Journal Articles

Detrimental Role of Excess Adenosine-Mediated 2,3-Diphosphoglycerate Induction in Erythrocyte Sickling and Novel Mechanism-Based Therapies.

Yujin Zhang, Yingbo Dai, Jiaming Wen, Rodney E. Kellems, Michael R Blackburn, Harinder S. Juneja, Yang Xia
Journal: Blood
Blood (2009) 114 (22): 906.
https://doi.org/10.1182/blood.V114.22.906.906
Published: 2009
Journal Articles

How I treat ADA deficiency

H. Bobby Gaspar, Alessandro Aiuti, Fulvio Porta, Fabio Candotti, Michael S. Hershfield, Luigi D. Notarangelo
Journal: Blood
Blood (2009) 114 (17): 3524–3532.
https://doi.org/10.1182/blood-2009-06-189209
Published: 2009
Journal Articles

Comparative Results of Gene Therapy for Adenosine Deaminase Deficiency with or without PEG-ADA Withdrawal and Myelosuppressive Chemotherapy.

Robert Sokolic, MD, Greg Podsakoff, MD, Linda Muul, PhD, Barbara Engel, MD, Jayashree Jagadeesh, BS, Elizabeth Garabedian, RN, MSLS, Denise Carbonaro, Laura Tuschong, BS, Joanna Ireland, Michael Hershfield, MD, John Tisdale, MD, Cynthia Dunbar, MD, Alan Wayne, MD, Donald Kohn, MD, Fabio Candotti, MD
Journal: Blood
Blood (2007) 110 (11): 501.
https://doi.org/10.1182/blood.V110.11.501.501
Published: 2007
Journal Articles

Long-Term Safety and Efficacy of Stem Cell Gene Therapy for ADA-SCID.

Alessandro Aiuti, Ulrike Benninghoff, Barbara Cassani, Federica Cattaneo, Luciano Callegaro, Grazia Andolfi, Massimiliano Mirolo, Samantha Scaramuzza, Sarah Marktel, Antonella Tabucchi, Filippo Carlucci, Martha Eibl, Memet Aker, Shimon Slavin, Fabio Ciceri, Roberto Miniero, Claudio Bordignon, Maria Grazia Roncarolo
Journal: Blood
Blood (2006) 108 (11): 200.
https://doi.org/10.1182/blood.V108.11.200.200
Published: 2006
Journal Articles

Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: long-term results of the first clinical gene therapy trial

Linda Mesler Muul, Laura M. Tuschong, Sherry Lau Soenen, G. Jayashree Jagadeesh, W. Jay Ramsey, Zhifeng Long, Charles S. Carter, Elizabeth K. Garabedian, Melinna Alleyne, Margaret Brown, Wendy Bernstein, Shepherd H. Schurman, Thomas A. Fleisher, Susan F. Leitman, Cynthia E. Dunbar, R. Michael Blaese, Fabio Candotti
Journal: Blood
Blood (2003) 101 (7): 2563–2569.
https://doi.org/10.1182/blood-2002-09-2800
Published: 2003
Journal Articles

Antibody responses to bacteriophage phi X174 in patients with adenosine deaminase deficiency

HD Ochs, RH Buckley, RH Kobayashi, AL Kobayashi, RU Sorensen, SD Douglas, BL Hamilton, MS Hershfield
Journal: Blood
Blood (1992) 80 (5): 1163–1171.
https://doi.org/10.1182/blood.V80.5.1163.1163
Published: 1992