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Augustine Fernandes
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Journal Articles

Ex Vivo Lentiviral Hematopoietic Stem Cell (HSC) Gene Therapy May Represent a Curative Therapy for the Life-Threatening Inborn Error of Immunity Severe Combined Immunodeficiency Due to Adenosine Deaminase (ADA) Deficiency (ADA-SCID)

Katelyn Masiuk, Jinhua Xu-Bayford, Augustine Fernandes, Konstantinos Vazouras, Danilo Pellin, Havinder Hara, Sohini Roy, Beatriz Campo Fernandez, Thao Thai Dang, Kimberly Gilmour, Elena Alvarez Mediavilla, Beatrice Curio-Penny, Gráinne O'Toole, Rima Ahmed, Jordyn Arnold, Elizabeth Garabedian, Harry L Malech, Kenneth Cornetta, Claire Booth, Donald B. Kohn
Journal: Blood
Blood (2024) 144 (Supplement 1): 1051.
https://doi.org/10.1182/blood-2024-202543
Published: 2024
Journal Articles

Patient and Parent-Reported Outcomes Post-Treatment with Shmir-Based Lentiviral Gene Therapy for Sickle Cell Disease

Lauren Jimenez-Kurlander, Pei-Chi Kao, Mira Magner, Emily Morris, Amy Federico, Theodore Moore, Satiro DeOliveira, Augustine Fernandes, Donald B. Kohn, Wendy B. London, David A. Williams, Erica B. Esrick
Journal: Blood
Blood (2023) 142 (Supplement 1): 3750.
https://doi.org/10.1182/blood-2023-186915
Published: 2023
Journal Articles

Interim Results from an Ongoing Phase 1/2 Study of Lentiviral-mediated Ex-vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)

Claire Booth, Julián Sevilla, Gayatri R. Rao, Maria Chitty Lopez, Elena Almarza, Dayna Terrazas, Josune Zubicaray, Marta González Vicent, Kritika Chetty, Grainne O'Toole, Jinhua Xu-Bayford, Eileen Nicoletti, Augustine Fernandes, Caroline Kuo, Satiro de Oliveira, Theodore B. Moore, Grace Choi, Miriam Zeini, Cristina Mesa-Núñez, Adrian J. Thrasher, Juan A. Bueren, Jonathan D. Schwartz, Donald B. Kohn
Journal: Blood
Blood (2022) 140 (Supplement 1): 7774–7775.
https://doi.org/10.1182/blood-2022-158578
Published: 2022
Journal Articles

Induction of Fetal Hemoglobin and Reduction of Clinical Manifestations in Patients with Severe Sickle Cell Disease Treated with Shmir-Based Lentiviral Gene Therapy for Post-Transcriptional Gene Editing of BCL11A: Updated Results from Pilot and Feasibility Trial

Erica B. Esrick, Amy Federico, Daniela Abriss, Myriam Armant, Kari Boardman, Christian Brendel, Marioara-Felicia Ciuculescu, Heather Daley, Colleen Dansereau, Augustine Fernandes, Matthew M. Heeney, David G. Justus, Pei-Chi Kao, Annette S. Kim, Donald B. Kohn, Leslie E. Lehmann, Donghui Liu, Wendy B. London, John P Manis, Theodore B. Moore, Emily Morris, Danilo Pellin, Ellen Proeung, Shanna Richard, Gavin D. Roach, Kit L. Shaw, Dayna Terrazas, Shanna L White, David A. Williams
Journal: Blood
Blood (2022) 140 (Supplement 1): 10665–10667.
https://doi.org/10.1182/blood-2022-169089
Published: 2022
Journal Articles

A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results

Donald B. Kohn, Claire Booth, Julián Sevilla, Gayatri R Rao, Elena Almarza, Dayna Terrazas, Eileen Nicoletti, Augustine Fernandes, Caroline Kuo, Satiro de Oliveira, Theodore B. Moore, Kenneth M Law, Brian C Beard, Grace Choi, Miriam Zeini, Cristina Mesa-Núñez, Adrian J. Thrasher, Juan A Bueren, Jonathan D Schwartz
Journal: Blood
Blood (2021) 138 (Supplement 1): 2932.
https://doi.org/10.1182/blood-2021-151898
Published: 2021
Journal Articles

Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency

Clinical Trials & Observations
Bryanna Reinhardt, Omar Habib, Kit L. Shaw, Elizabeth Garabedian, Denise A. Carbonaro-Sarracino, Dayna Terrazas, Beatriz Campo Fernandez, Satiro De Oliveira, Theodore B. Moore, Alan K. Ikeda, Barbara C. Engel, Gregory M. Podsakoff, Roger P. Hollis, Augustine Fernandes, Connie Jackson, Sally Shupien, Suparna Mishra, Alejandra Davila, Jack Mottahedeh, Andrej Vitomirov, Wenzhao Meng, Aaron M. Rosenfeld, Aoife M. Roche, Pascha Hokama, Shantan Reddy, John Everett, Xiaoyan Wang, Eline T. Luning Prak, Kenneth Cornetta, Michael S. Hershfield, Robert Sokolic, Suk See De Ravin, Harry L. Malech, Frederic D. Bushman, Fabio Candotti, Donald B. Kohn
Journal: Blood
Blood (2021) 138 (15): 1304–1316.
https://doi.org/10.1182/blood.2020010260
Published: 2021
Includes: Supplemental data
Journal Articles

A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1

Donald B. Kohn, MD, Gayatri R Rao, MD JD, Elena Almarza, PhD, Dayna Terrazas, RN, Eileen Nicoletti, MD, Augustine Fernandes, PhD, Caroline Kuo, MD, Satiro N. De Oliveira, MD, Theodore B. Moore, MD, Kenneth M Law, PhD, Brian C Beard, PhD, Julian Sevilla, MD PhD, Cristina Mesa-Núñez, PhD, Juan A Bueren, PhD, Jonathan D Schwartz, MD
Journal: Blood
Blood (2020) 136 (Supplement 1): 15.
https://doi.org/10.1182/blood-2020-142484
Published: 2020
Journal Articles

The molecular basis of hepcidin-resistant hereditary hemochromatosis

Augustine Fernandes, Gloria C. Preza, Yen Phung, Ivana De Domenico, Jerry Kaplan, Tomas Ganz, Elizabeta Nemeth
Journal: Blood
Blood (2009) 114 (2): 437–443.
https://doi.org/10.1182/blood-2008-03-146134
Published: 2009
Includes: Supplemental data
Journal Articles

Structural Aspects of Hepcidin-Ferroportin Binding

Gloria Preza, Augustine Fernandes, PhD, Richard J. Clark, PhD, David J. Craik, PhD, Tomas Ganz, MD, PhD, Elizabeta Nemeth, PhD
Journal: Blood
Blood (2008) 112 (11): 119.
https://doi.org/10.1182/blood.V112.11.119.119
Published: 2008