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Jonathan D Schwartz, MD
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Journal Articles

Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: A Global Phase 1 Study for Adult and Pediatric Patients

José Luis López Lorenzo, MD, Susana Navarro, PhD, Ami J Shah, MD, Maria Grazia Roncarolo, MD, Julián Sevilla, MD PhD, Lucía Llanos, MD PhD, Begoña Pérez Camino de Gaisse, MD, Sol Sanchez, MD, Bertil Glader, MD PhD, May Chien, MD, Oscar Quintana Bustamante, PhD, Brian C Beard, PhD, Kenneth M Law, PhD, Miriam Zeini, PhD, Grace Choi, BS, Eileen Nicoletti, MD, Juan A Bueren, PhD, Gayatri R Rao, MD JD, Jonathan D Schwartz, MD, José C Segovia, PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 47.
https://doi.org/10.1182/blood-2020-137246
Published: 2020
Journal Articles

Gene Therapy for Fanconi Anemia, Complementation Group a: Updated Results from Ongoing Global Clinical Studies of RP-L102

Agnieszka Czechowicz, MDPhD, Rajni Agarwal, MD, Julián Sevilla, MD PhD, Paula Río, PhD, Susana Navarro, PhD, Brian C Beard, PhD, Kenneth M Law, PhD, Grace Choi, BS, Miriam Zeini, PhD, Eileen Nicoletti, MD, John E Wagner, MD, Claire Booth, MBBS, PhD MSc, Jonathan D Schwartz, MD, Juan A Bueren, PhD, Maria Grazia Roncarolo, MD
Journal: Blood
Blood (2020) 136 (Supplement 1): 14.
https://doi.org/10.1182/blood-2020-137106
Published: 2020
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

Gene Therapy for Lad-I Immunodeficiency: Preclinical Evaluation of HSC Transduction Under Optimized GMP-Conditions

Elena Almarza, PhD, Cristina Mesa-Núñez, PhD, Carlos Damián, María Fernández-García, Begoña Díez-Cabezas, PhD, M Luz Lozano, Paula Rio, PhD, Rosa M Yañez, PhD, Ken Law, PhD, Brian C Beard, PhD, Jonathan D Schwartz, MD, Juan A. Bueren, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 5751.
https://doi.org/10.1182/blood-2019-122878
Published: 2019
Journal Articles

Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene Therapy

Paula Rio, PhD, Susana Navarro, PhD, Rebeca Sanchez-Dominguez, PhD, Jose C Segovia, PhD, Wei Wang, PhD, Eva Merino, Rosa M Yañez, PhD, Jose Antonio Casado, PhD, Yari A. Gimenez, Francisco J Roman-Rodriguez, PhD, Omaira Alberquilla, Eva Galvez, MD, Raquel Hladun, MD, Jordi Barquinero, PhD, Anne Galy, PharmD, PhD, Nagore García de Andoín, MD, Ricardo López, MD PhD, Albert Catalá, MD PhD, Marina Cavazzana, Francois Lefrere, MD, Thierry Leblanc, MD, Jonathan D Schwartz, MD, Roser Pujol, PhD, Jordi Surrallés, PhD, Jean Soulier, MD PhD, Manfred Schmidt, PhD, Cristina Díaz de Heredia, MD PhD, Julian Sevilla, MD PhD, Juan A. Bueren, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4627.
https://doi.org/10.1182/blood-2019-129686
Published: 2019
Journal Articles

Changing the Natural History of Fanconi Anemia Complementation Group-A with Gene Therapy: Early Results of U.S. Phase I Study of Lentiviral-Mediated Ex-Vivo FANCA Gene Insertion in Human Stem and Progenitor Cells

Agnieszka Czechowicz, MD PhD, Maria Grazia Roncarolo, MD, Brian C Beard, PhD, Ken Law, PhD, Eileen Nicoletti, MD, Paula Río, PhD, Juan A. Bueren, PhD, Jonathan D Schwartz, MD, Sandeep Soni, MBBS
Journal: Blood
Blood (2019) 134 (Supplement_1): 3350.
https://doi.org/10.1182/blood-2019-127352
Published: 2019
Journal Articles

Preclinical Evaluation for the Gene Therapy of Patients with Leukocyte Adhesion Deficiency Type I

Elena Almarza, PhD, Cristina Mesa-Núñez, Carlos Damián, María Fernández-García, Begoña Díez-Cabezas, Jonathan D Schwartz, MD, Juan A Bueren
Journal: Blood
Blood (2018) 132 (Supplement 1): 5798.
https://doi.org/10.1182/blood-2018-99-111136
Published: 2018
Journal Articles

Advances in the Gene Therapy of Patients with Fanconi Anemia

Juan A. Bueren, PhD, Susana Navarro, PhD, Wei Wang, PhD, Rebeca Sanchez-Dominguez, PhD, Eva Merino, Jose C Segovia, PhD, Anne Galy, PharmD, PhD, María L. Lamana, MD PhD, Rosa M Yañez, PhD, Jose A Casado, PhD, Yari A. Gimenez, Francisco J Roman-Rodriguez, Lara A. Alvarez, Roser Pujol, PhD, Jordi Surralles, PhD, Ricardo López, MD PhD, Nagore García de Andoín, MD, Albert Catala, MD PhD, Eva Galvez, MD, Raquel Hladun, MD, Jonathan D Schwartz, MD, Manfred Schmidt, PhD, Cristina Díaz de Heredia, MD PhD, Paula Rio, PhD, Julian Sevilla, MD PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 1022.
https://doi.org/10.1182/blood-2018-99-111160
Published: 2018
Journal Articles

Leukocyte Adhesion Deficiency-I (LAD-I): A Comprehensive Review of Published Cases

Sanchali Kasbekar, PharmD, Juan Bueren, PhD, Adrian J Thrasher, MD PhD FMedSci, Donald B Kohn, MD, Julian Sevilla, MD PhD, Jonathan D Schwartz, MD, Elena Almarza, PhD
Journal: Blood
Blood (2017) 130 (Supplement 1): 4821.
https://doi.org/10.1182/blood.V130.Suppl_1.4821.4821
Published: 2017
Journal Articles

SL-801, a Novel, Reversible Inhibitor of Exportin-1 (XPO1) / Chromosome Region Maintenance-1 (CRM1) with Broad and Potent Anti-Cancer Activity

Janice Chen, PhD, Christopher L. Brooks, PhD, Peter McDonald, Jonathan D. Schwartz, MD, Rebecca S. Schneider, Keiichi Sakakibara, PhD, Naoya Saito, PhD, Takuji Sato, MSc, Takumi Kawabe, MD PhD, Eric K. Rowinsky, MD
Journal: Blood
Blood (2015) 126 (23): 4433.
https://doi.org/10.1182/blood.V126.23.4433.4433
Published: 2015
Journal Articles

Lead-in Stage Results of a Pivotal Trial of SL-401, an Interleukin-3 Receptor (IL-3R) Targeting Biologic, in Patients with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) or Acute Myeloid Leukemia (AML)

Kendra L. Sweet, MD, Naveen Pemmaraju, MD, Andrew A Lane, MD PhD, Anthony S. Stein, MD, Sumithira Vasu, MBBS, William Blum, MD, David A. Rizzieri, MD, Eunice S. Wang, MD, Eric K. Rowinsky, MD, Michael Szarek, PhD, Christopher L. Brooks, PhD, Sarah Disalvatore, MPH, David Liu, Madeleine Duvic, MD, Jonathan D. Schwartz, MD, Marina Konopleva, MD PhD
Journal: Blood
Blood (2015) 126 (23): 3795.
https://doi.org/10.1182/blood.V126.23.3795.3795
Published: 2015