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Journal Articles

Bone Marrow Failure Associated with Short Telomeres and Digenic Variants of Uncertain Significance in Telomere Biology Genes

Akhila Vadivelan, Julian Martinez, Satiro De Oliveira, Theodore Moore, Alan Ikeda, Geraldine Aubert, Vivian Y. Chang
Journal: Blood
Blood (2023) 142 (Supplement 1): 5669.
https://doi.org/10.1182/blood-2023-177530
Published: 2023
Journal Articles

Long-Term Outcome of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1) Using an Enhancer-Deleted Self-Inactivating Gammaretroviral Vector

Sung-Yun Pai, Nisha Nagarsheth, Susan Prockop, Myriam Armant, Donald B. Kohn, Rebecca A. Marsh, Claire Booth, John K. Everett, Jack Bleesing, Deepak Chellapandian, Colleen Dansereau, Satiro de Oliveira, Wendy B. London, M. Angélica Marinovic, Theodore B. Moore, Matias Oleastro, Grainne O'Toole, Mark Vander Lugt, Luciano Urdinez, Kelly J. Walkovich, Jolan E. Walter, Axel Schambach, Adrian J. Thrasher, Frederic D. Bushman, David A Williams
Journal: Blood
Blood (2022) 140 (Supplement 1): 10643–10645.
https://doi.org/10.1182/blood-2022-159768
Published: 2022
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

Lentiviral Gene Therapy with Low Dose Conditioning for X-Linked SCID Results in Complete Immune Reconstitution and No Evidence of Clonal Expansion

Claire Booth, Donald B. Kohn, Myriam Armant, Susan Prockop, Karen Buckland, Sharat Chandra, Shanmuganathan Chandrakasan, Kritika Chetty, Colleen Dansereau, Satiro de Oliveira, John Everett, Diego León Rico, Wendy B. London, Jin Hua Xu-Bayford, Theodore B. Moore, Nisha Nagarsheth, Suhag Parikh, Dayna Terrazas, Shanna L White, Axel Schambach, Frederic D. Bushman, Adrian J. Thrasher, David A. Williams, Sung-Yun Pai
Journal: Blood
Blood (2022) 140 (Supplement 1): 7770–7771.
https://doi.org/10.1182/blood-2022-159979
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

First Report of Non-Genotoxic Conditioning with JSP191 (anti-CD117) and Hematopoietic Stem Cell Transplantation in a Newly Diagnosed Patient with Severe Combined Immune Deficiency

Rajni Agarwal, MD, Kenneth I. Weinberg, MD, Hye-Sook Kwon, PhD, Anne Le, Janel R Long-Boyle, PharmD, PhD, Donald B. Kohn, MD, Kathryn Bradford, MD, Satiro De Oliveira, MD, Alice Bertaina, MD PhD, Agnieszka Czechowicz, MDPhD, Matt Porteus, MD PhD, David C Shyr, MD, Katja G Weinacht, MD PhD, Ami J Shah, MD, Melissa Mavers, MD PhD, Elisabeth Merkel, RN, Janice W Brown, MD, Christopher C. Dvorak, MD, Robertson Parkman, MD, Maria Grazia Roncarolo, MD, Judith A Shizuru, MD PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 10.
https://doi.org/10.1182/blood-2020-137762
Published: 2020