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Journal Articles

Analysis of Deficiency of Adenosine Deaminase 2 Pathogenesis Based on Single Cell RNA Sequencing of Monocytes

Naoki Watanabe, Shouguo Gao, PhD, Sachiko Kajigaya, Carrie Diamond, MD, Lemlem Alemu, BS, Amanda Ombrello, MD, Neal S. Young, MD

Blood (2019) 134 (Supplement_1): 2317.

https://doi.org/10.1182/blood-2019-123859

Published: 2019

Journal Articles

Modeling of Immune Reconstitution Post CD34 Selected Stem Cell Transplantation in Pediatric Patients with Severe Combined Immune Deficiency

Jean-Sebastien Diana, MD, Naim Bouazza, PhD, Chloé Couzin, MD, Martin Castelle, MD, Alessandra Magnani, MD PhD, Elisa Magrin, PhD, Laure Joseph, MD, Marianne Delville, MD, Francois Lefrere, MD, jean-Marc Treluyer, MD PhD, Capucine Picard, MD PhD, Despina Moshous, MD PhD, Isabelle André, PhD, Stephane Blanche, MD, Benedicte Neven, MD PhD, Marina Cavazzana, MD PhD

Blood (2019) 134 (Supplement_1): 5668.

https://doi.org/10.1182/blood-2019-127974

Published: 2019

Journal Articles

Survival Outcomes Following Allogeneic Stem Cell Transplantation for Inherited Bone Marrow Failure and Myeloid Germline Predisposition Syndromes

Kalyan Nadiminti, Abhishek A Mangaonkar, MBBS, Kimberly J. Langer, D.N.P., C.N.P., Shakila P Khan, MD, Vilmarie Rodriguez, MD, Naseema Gangat, MBBS, Michelle A Elliott, MD, Hassan B. Alkhateeb, MD, Mithun Vinod Shah, MD PhD, Mark Litzow, MD, William J Hogan, MB, BCh, Saad S. Kenderian, MD, Mrinal M Patnaik, MDMBBS

Blood (2019) 134 (Supplement_1): 3300.

https://doi.org/10.1182/blood-2019-121742

Published: 2019

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

Blood (2019) 134 (Supplement_1): 3345.

https://doi.org/10.1182/blood-2019-123432

Published: 2019

Journal Articles

Deficiency of adenosine deaminase 2 triggers adenosine-mediated NETosis and TNF production in patients with DADA2

Carmelo Carmona-Rivera, Sami S. Khaznadar, Kyawt W. Shwin, Jorge A. Irizarry-Caro, Liam J. O’Neil, Yudong Liu, Kenneth A. Jacobson, Amanda K. Ombrello, Deborah L. Stone, Wanxia L. Tsai, Daniel L. Kastner, Ivona Aksentijevich, Mariana J. Kaplan, Peter C. Grayson

Blood (2019) 134 (4): 395–406.

https://doi.org/10.1182/blood.2018892752

Published: 2019

Includes: Supplemental data

Journal Articles

Deficiency of Adenosine Deaminase 2 (DADA2) Presenting As Familial Hodgkin Lymphoma

Fahad Alabbas, Omar Alsharief, Isabelle Meyts, Fatma Albatniji, Michael Hershfield, Adnan Mansoor, MD PhD, Ghaleb Elyamany, MD PhD

Blood (2018) 132 (Supplement 1): 5373.

https://doi.org/10.1182/blood-2018-99-116431

Published: 2018

Journal Articles

Umbilical Cord Blood Mononuclear Cells for Ex-Vivo Gene Therapy

Farid V. Bashirov, Ilnur I. Salafutdinov, PhD, Michail E. Sokolov, Andrew A. Izmailov, MD, Vage A. Markosyan, Filip O. Fadeev, Albert Rizvanov, DSc, PhD, Rustem I. Islamov

Blood (2018) 132 (Supplement 1): 5797.

https://doi.org/10.1182/blood-2018-99-113462

Published: 2018

Journal Articles

Hematopoietic stem cell transplantation rescues the hematological, immunological, and vascular phenotype in DADA2

Blood (2017) 130 (24): 2682–2688.

https://doi.org/10.1182/blood-2017-07-798660

Published: 2017

Journal Articles

Diamond-Blackfan Anemia Phenotype Caused By Deficiency of Adenosine Deaminase 2

Blood (2017) 130 (Supplement 1): 874.

https://doi.org/10.1182/blood.V130.Suppl_1.874.874

Published: 2017

Journal Articles

Cicalese MP, Ferrua F, Castagnaro L, et al. Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency. Blood. 2016;128(1):45-54.

Blood (2017) 129 (24): 3271.

https://doi.org/10.1182/blood-2017-05-782862

Published: 2017

Journal Articles

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

Blood (2016) 128 (1): 45–54.

https://doi.org/10.1182/blood-2016-01-688226

Published: 2016

Includes: Supplemental data

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

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

Blood (2011) 118 (21): 4556.

https://doi.org/10.1182/blood.V118.21.4556.4556

Published: 2011

Journal Articles

Myeloid dysplasia and bone marrow hypocellularity in adenosine deaminase-deficient severe combined immune deficiency

Robert Sokolic, Irina Maric, Chimene Kesserwan, Elizabeth Garabedian, I. Celine Hanson, Margaret Dodds, Rebecca Buckley, Andrew C. Issekutz, Naynesh Kamani, Kit Shaw, Ben Tan, Pawan Bali, Michael S. Hershfield, Donald B. Kohn, Alan S. Wayne, Fabio Candotti

Blood (2011) 118 (10): 2688–2694.

https://doi.org/10.1182/blood-2011-01-329359

Published: 2011

Journal Articles

How I treat ADA deficiency

H. Bobby Gaspar, Alessandro Aiuti, Fulvio Porta, Fabio Candotti, Michael S. Hershfield, Luigi D. Notarangelo

Blood (2009) 114 (17): 3524–3532.

https://doi.org/10.1182/blood-2009-06-189209

Published: 2009

Journal Articles

ADA-deficient SCID is associated with a specific microenvironment and bone phenotype characterized by RANKL/OPG imbalance and osteoblast insufficiency

Aisha V. Sauer, Emanuela Mrak, Raisa Jofra Hernandez, Elena Zacchi, Francesco Cavani, Miriam Casiraghi, Eyal Grunebaum, Chaim M. Roifman, Maria C. Cervi, Alessandro Ambrosi, Filippo Carlucci, Maria Grazia Roncarolo, Anna Villa, Alessandro Rubinacci, Alessandro Aiuti

Blood (2009) 114 (15): 3216–3226.

https://doi.org/10.1182/blood-2009-03-209221

Published: 2009

Includes: Supplemental data

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

Blood (2007) 110 (11): 501.

https://doi.org/10.1182/blood.V110.11.501.501

Published: 2007

Journal Articles

Patients with adenosine deaminase deficiency surviving after hematopoietic stem cell transplantation are at high risk of CNS complications

Manfred Hönig, Michael H. Albert, Ansgar Schulz, Monika Sparber-Sauer, Catharina Schütz, Bernd Belohradsky, Tayfun Güngör, Markus T. Rojewski, Harald Bode, Ulrich Pannicke, Dominique Lippold, Klaus Schwarz, Klaus-Michael Debatin, Michael S. Hershfield, Wilhelm Friedrich

Blood (2006) 109 (8): 3595–3602.

https://doi.org/10.1182/blood-2006-07-034678

Published: 2006

Journal Articles

Deoxyadenosine Inhibits T-Cell Activation in ADA-SCID Patients through a cAMP/PKA-Dependent Pathway.

Barbara Cassani, Massimiliano Mirolo, Federica Cattaneo, Ulrike Benninghoff, Filippo Carlucci, Antonella Tabucchi, Claudio Bordignon, Maria Grazia Roncarolo, Alessandro Aiuti

Blood (2006) 108 (11): 1250.

https://doi.org/10.1182/blood.V108.11.1250.1250

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

Blood (2003) 101 (7): 2563–2569.

https://doi.org/10.1182/blood-2002-09-2800

Published: 2003

NARROW

Analysis of Deficiency of Adenosine Deaminase 2 Pathogenesis Based on Single Cell RNA Sequencing of Monocytes

Modeling of Immune Reconstitution Post CD34 Selected Stem Cell Transplantation in Pediatric Patients with Severe Combined Immune Deficiency

Survival Outcomes Following Allogeneic Stem Cell Transplantation for Inherited Bone Marrow Failure and Myeloid Germline Predisposition Syndromes

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

Deficiency of adenosine deaminase 2 triggers adenosine-mediated NETosis and TNF production in patients with DADA2

Deficiency of Adenosine Deaminase 2 (DADA2) Presenting As Familial Hodgkin Lymphoma

Umbilical Cord Blood Mononuclear Cells for Ex-Vivo Gene Therapy

Hematopoietic stem cell transplantation rescues the hematological, immunological, and vascular phenotype in DADA2

Diamond-Blackfan Anemia Phenotype Caused By Deficiency of Adenosine Deaminase 2

Cicalese MP, Ferrua F, Castagnaro L, et al. Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency. Blood. 2016;128(1):45-54.

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

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

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

Myeloid dysplasia and bone marrow hypocellularity in adenosine deaminase-deficient severe combined immune deficiency

How I treat ADA deficiency

ADA-deficient SCID is associated with a specific microenvironment and bone phenotype characterized by RANKL/OPG imbalance and osteoblast insufficiency

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

Patients with adenosine deaminase deficiency surviving after hematopoietic stem cell transplantation are at high risk of CNS complications

Deoxyadenosine Inhibits T-Cell Activation in ADA-SCID Patients through a cAMP/PKA-Dependent Pathway.

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

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American Society of Hematology

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ASH Publications

American Society of Hematology

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