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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

Establishment of Novel Leukemic Cell Lines Resistant to Clofarabine by Dual Mechanisms of Decreased Active Metabolite and Increased Antiapoptotic Factor

Hiroko Shigemi, MD, Takahiro Yamauchi, MD, PhD, Takanori Ueda, MD, PhD

Blood (2012) 120 (21): 1356.

https://doi.org/10.1182/blood.V120.21.1356.1356

Published: 2012

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

Frontline Therapy for Older Patients (pts) with Acute Myeloid Leukemia (AML): Clofarabine Plus Low-Dose Cytarabine Induction Followed by Prolonged Consolidation with Clofarabine Plus Low-Dose Cytarabine Alternating with Decitabine

Stefan Faderl, MD, Farhad Ravandi, MD, Guillermo Garcia-Manero, MD, Xuelin Huang, PhD, Elias Jabbour, MD, Tapan Kadia, MD, Alessandra Ferrajoli, MD, Zeev Estrov, MD, Jennie Feliu, RN, Heather Schroeder, RN, Monica Kwari, BSN, Hagop Kantarjian, MD

Blood (2010) 116 (21): 336.

https://doi.org/10.1182/blood.V116.21.336.336

Published: 2010

Journal Articles

Comparative in-Vitro Evaluation of the Myeloid Toxicity of Pentostatin and the Novel PNP-Inhibitor Forodesine.

Mario Schubert, MD, Christian Wallenwein, Larissa Pietsch, Dan Ran, Isabel Taubert, Katrin Miesala, Patrick Wuchter, MD, Volker Eckstein, PhD, Anthony D. Ho, MD, PhD

Blood (2009) 114 (22): 3766.

https://doi.org/10.1182/blood.V114.22.3766.3766

Published: 2009

Journal Articles

Oral Clofarabine in the Treatment of Patients with Higher-Risk Myelodysplastic Syndrome.

Stefan Faderl, MD, Guillermo Garcia-Manero, MD, Zeev Estrov, MD, Farhad Ravandi, MD, Gautam Borthakur, MD, Jorge Cortes, MD, Susan O'Brien, MD, Varsha Gandhi, PhD, William Plunkett, PhD, Anna L. Byrd, RN, Monica Kwari, RN, Hagop Kantarjian, MD

Blood (2009) 114 (22): 118.

https://doi.org/10.1182/blood.V114.22.118.118

Published: 2009

Journal Articles

Clofarabine Plus Low-Dose Cytarabine Induction Followed by Consolidation with Clofarabine Plus Low-Dose Cytarabine Alternating with Decitabine as Frontline Therapy for Patients (pts) with Acute Myeloid Leukemia (AML) ≥ 60 Years (yrs).

Sameer A Parikh, MD, Hagop Kantarjian, M.D., Guillermo Garcia-Manero, MD, Elias Jabbour, MD, Tapan Kadia, MD, Farhad Ravandi, MD, Alessandra Ferrajoli, MD, Zeev Estrov, MD, Jenny Feliu, RN, Stefan Faderl, MD

Blood (2009) 114 (22): 2058.

https://doi.org/10.1182/blood.V114.22.2058.2058

Published: 2009

Journal Articles

Results of a Randomized Phase 2 Study of Clofarabine Plus Cytarabine (CA) Vs Clofarabine Plus Idarubicin (CI) Vs Clofarabine, Idarubicin, Plus Cytarabine (CIA) as Salvage Therapy in Acute Myeloid Leukemia (AML).

Stefan Faderl, Gautam Borthakur, Farhad Ravandi, Xuelin Huang, Miloslav Beran, Alessandra Ferrajoli, Charles A. Koller, Deborah A. Thomas, Srdan Verstovsek, Monica Kwari, Hagop M. Kantarjian

Blood (2008) 112 (11): 964.

https://doi.org/10.1182/blood.V112.11.964.964

Published: 2008

Journal Articles

Oral (po) and Intravenous (iv) Clofarabine for Patients (pts) with Myelodysplastic Syndrome (MDS)

Stefan Faderl, Guillermo Garcia-Manero, Farhad Ravandi, Gautam Borthakur, Zeev Estrov, Deborah A. Thomas, Varsha Gandhi, William Plunkett, Anna Byrd, Monica Kwari, Hagop M. Kantarjian

Blood (2008) 112 (11): 222.

https://doi.org/10.1182/blood.V112.11.222.222

Published: 2008

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

Valproate, a Histone Deacetylase Inhibitor, Enhances Purine Nucleoside Analogues Induced Apoptosis of B-Chronic Lymphocytic Leukemia Cells.

Amel B. Bouzar, PhD, Mathieu Boxus, MS, Julien Defoiche, MS, Hervé Balon Techn, Guy Berchem, PhD, Derek Macallan, PhD, Ruth Pettengel, PhD, Fenella Willis, PhD, Arsene Burny, PhD, Laurence Lagneaux, PhD, Dominique Bron, PhD, Bernard Chatelain, PhD, Christian Chatelain, PhD, Luc Willems, PhD

Blood (2007) 110 (11): 4712.

https://doi.org/10.1182/blood.V110.11.4712.4712

Published: 2007

Journal Articles

Mechanism for the Immuno-Suppresion Activity of Pentostatin: Selective Apoptosis of Naive T Cell Subsets.

Melody M. Smith, BS, William Powers, Cindy R. Giver, Ph.D., Ned Waller, M.D. Ph.D, Christopher Flowers, M.D.

Blood (2006) 108 (11): 5160.

https://doi.org/10.1182/blood.V108.11.5160.5160

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

Phase II Study of Clofarabine and Cytosine Arabinoside in Adult Patients with Relapsed AML and in Elderly Patients with Untreated AML Who Are at High Risk of Anthracycline Toxicity.

Edward Agura, MD, R. Brian Berryman, MD, Laura Brougher, RN, Barry Cooper, MD, Andrew De Leon, RPh, Joseph Fay, MD, Houston Holmes, MD, Christopher Maisel, MD, Arturo Molina, MD, Luis Pineiro, MD, Giovanna Saracino, MD, Mirjana Tadic-Ovcina, MD, Estil Vance, MD

Blood (2006) 108 (11): 1951.

https://doi.org/10.1182/blood.V108.11.1951.1951

Published: 2006

Journal Articles

Clofarabine and Clofarabine Plus Low-Dose Cytarabine (ara-C) as Induction Therapy for Patients (pts) ≥ 60 Years with Newly Diagnosed Acute Myeloid Leukemia (AML).

Stefan Faderl, MD, Farhad Ravandi, MD, Alessandra Ferrajoli, MD, Guillermo Garcia-Manero, MD, Zeev Estrov, MD, Deborah A. Thomas, MD, Susan O’Brien, MD, Srdan Verstovsek, MD, PhD, William Wierda, MD, PhD, Jorge Cortes, MD, Francis J. Giles, MD, Monica Kwari, R.N., Rita Jarmon, R.N., Hagop M. Kantarjian, MD

Blood (2005) 106 (11): 2804.

https://doi.org/10.1182/blood.V106.11.2804.2804

Published: 2005

Journal Articles

Neutrophil proPR3 Induces S-Phase Arrest in Myeloid Progenitors by Inhibition of Ribonucleotide Reductase Mediated by Nitric Oxide.

Tor Olofsson, PhD, Stefan Skold, MD, PhD, Urban Gullberg, PhD

Blood (2004) 104 (11): 1269.

https://doi.org/10.1182/blood.V104.11.1269.1269

Published: 2004

Journal Articles

Adenosine deaminase deficiency with mosaicism for a “second-site suppressor” of a splicing mutation: decline in revertant T lymphocytes during enzyme replacement therapy

Francisco X. Arredondo-Vega, Ines Santisteban, Eva Richard, Pawan Bali, Majed Koleilat, Michael Loubser, Abdulaziz Al-Ghonaium, Mariam Al-Helali, Michael S. Hershfield

Blood (2002) 99 (3): 1005–1013.

https://doi.org/10.1182/blood.V99.3.1005

Published: 2002

Journal Articles

Deoxyadenosine analogs induce programmed cell death in chronic lymphocytic leukemia cells by damaging the DNA and by directly affecting the mitochondria

Davide Genini, Souichi Adachi, Qi Chao, David W. Rose, Carlos J. Carrera, Howard B. Cottam, Dennis A. Carson, Lorenzo M. Leoni

Blood (2000) 96 (10): 3537–3543.

https://doi.org/10.1182/blood.V96.10.3537

Published: 2000

Journal Articles

Human Monocytoid Leukemia Cells Are Highly Sensitive to Apoptosis Induced by 2′-Deoxycoformycin and 2′-Deoxyadenosine: Association With dATP-Dependent Activation of Caspase-3

Nozomi Niitsu, Yuri Yamaguchi, Masanori Umeda, Yoshio Honma

Blood (1998) 92 (9): 3368–3375.

https://doi.org/10.1182/blood.V92.9.3368

Published: 1998

Journal Articles

Adenosine Deaminase Deficiency in Adults

Hulya Ozsahin, Francisco X. Arredondo-Vega, Ines Santisteban, Hanspeter Fuhrer, Peter Tuchschmid, Wolfram Jochum, Adriano Aguzzi, Howard M. Lederman, Amy Fleischman, Jerry A. Winkelstein, Reinhard A. Seger, Michael S. Hershfield

Blood (1997) 89 (8): 2849–2855.

https://doi.org/10.1182/blood.V89.8.2849

Published: 1997

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

Establishment of Novel Leukemic Cell Lines Resistant to Clofarabine by Dual Mechanisms of Decreased Active Metabolite and Increased Antiapoptotic Factor

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

Frontline Therapy for Older Patients (pts) with Acute Myeloid Leukemia (AML): Clofarabine Plus Low-Dose Cytarabine Induction Followed by Prolonged Consolidation with Clofarabine Plus Low-Dose Cytarabine Alternating with Decitabine

Comparative in-Vitro Evaluation of the Myeloid Toxicity of Pentostatin and the Novel PNP-Inhibitor Forodesine.

Oral Clofarabine in the Treatment of Patients with Higher-Risk Myelodysplastic Syndrome.

Clofarabine Plus Low-Dose Cytarabine Induction Followed by Consolidation with Clofarabine Plus Low-Dose Cytarabine Alternating with Decitabine as Frontline Therapy for Patients (pts) with Acute Myeloid Leukemia (AML) ≥ 60 Years (yrs).

Results of a Randomized Phase 2 Study of Clofarabine Plus Cytarabine (CA) Vs Clofarabine Plus Idarubicin (CI) Vs Clofarabine, Idarubicin, Plus Cytarabine (CIA) as Salvage Therapy in Acute Myeloid Leukemia (AML).

Oral (po) and Intravenous (iv) Clofarabine for Patients (pts) with Myelodysplastic Syndrome (MDS)

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

Valproate, a Histone Deacetylase Inhibitor, Enhances Purine Nucleoside Analogues Induced Apoptosis of B-Chronic Lymphocytic Leukemia Cells.

Mechanism for the Immuno-Suppresion Activity of Pentostatin: Selective Apoptosis of Naive T Cell Subsets.

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

Phase II Study of Clofarabine and Cytosine Arabinoside in Adult Patients with Relapsed AML and in Elderly Patients with Untreated AML Who Are at High Risk of Anthracycline Toxicity.

Clofarabine and Clofarabine Plus Low-Dose Cytarabine (ara-C) as Induction Therapy for Patients (pts) ≥ 60 Years with Newly Diagnosed Acute Myeloid Leukemia (AML).

Neutrophil proPR3 Induces S-Phase Arrest in Myeloid Progenitors by Inhibition of Ribonucleotide Reductase Mediated by Nitric Oxide.

Adenosine deaminase deficiency with mosaicism for a “second-site suppressor” of a splicing mutation: decline in revertant T lymphocytes during enzyme replacement therapy

Deoxyadenosine analogs induce programmed cell death in chronic lymphocytic leukemia cells by damaging the DNA and by directly affecting the mitochondria

Human Monocytoid Leukemia Cells Are Highly Sensitive to Apoptosis Induced by 2′-Deoxycoformycin and 2′-Deoxyadenosine: Association With dATP-Dependent Activation of Caspase-3

Adenosine Deaminase Deficiency in Adults

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