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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

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

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

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

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

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

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

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

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

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

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

Blood (1992) 80 (5): 1163–1171.

https://doi.org/10.1182/blood.V80.5.1163.1163

Published: 1992

NARROW

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

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

Adenosine Signaling in Priapism and Novel Therapies

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

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

How I treat ADA deficiency

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

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

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

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

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American Society of Hematology

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American Society of Hematology

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