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José C Segovia, PhD
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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

Modelling Congenital Dyserythropoietic Anemia Type II through Gene Editing in Hematopoietic Stem and Progenitor Cells

Mercedes Dessy-Rodriguez, Sara Fañanas-Baquero, Veronica Venturi, Salvador Payán-Pernía, Cristian Tornador, Gonzalo Hernandez, Mayka Sanchez, José C Segovia, PhD, Oscar Quintana Bustamante, PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 27.
https://doi.org/10.1182/blood-2020-139207
Published: 2020
Journal Articles

Correction of the Energetic Defects in Pyruvate Kinase Deficiency through Genome Editing in Hematopoietic Stem and Progenitor Cells

Sara Fañanas-Baquero, Oscar Quintana Bustamante, PhD, Daniel P. Dever, PhD, Omaira Alberquilla, Rebeca Sanchez-Dominguez, PhD, Joab Camarena, BS, Rolf Turk, José Luis López Lorenzo, MD, Paola Bianchi, BSc, PhD, Mark Behlke, MD PhD, Juan A Bueren, PhD, Matt Porteus, MD PhD, José C Segovia, PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 29.
https://doi.org/10.1182/blood-2020-139117
Published: 2020
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

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

Efficient CRISPR/Cas9-Mediated Gene Editing of Pklr in Human Hematopoietic Progenitors and Stem Cells for the Gene Therapy of Pyruvate Kinase Deficiency

Oscar Quintana Bustamante, PhD, Sara Fañanas-Baquero, Daniel P. Dever, PhD, Alberquilla Omaira, Joab Camarena, BS, Rebeca Sanchez-Dominguez, PhD, Matias Morin, Val Fernandez, Miguel Angel Moreno-Pelayo, Juan A. Bueren, PhD, Matthew Porteus, MD PhD, Jose C Segovia, PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 5792.
https://doi.org/10.1182/blood-2018-99-111772
Published: 2018
Journal Articles

Estrogens Enhance Human Hematopoietic Stem Cell Engraftment in a Xenogenic Transplantation Model

Sara Fañanas-Baquero, Israel Orman, Federico Becerra Aparicio, Silvia Bermudez de Miguel, Jordi Garcia Merino, Rosa M Yañez, PhD, Yolanda Fernandez, David Alfaro, Agustin Zapata, Juan A. Bueren, PhD, Jose C Segovia, PhD, Oscar Quintana Bustamante, PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 2042.
https://doi.org/10.1182/blood-2018-99-112104
Published: 2018
Journal Articles

Gene Editing of the Human Pklr Gene in Human Hematopoietic Progenitors to Correct Pyruvate Kinase Deficiency

Oscar Quintana-Bustamante, Sara Fañanas, Israel Orman, Agnès Gouble, PhD, Roman Galetto, PhD, Laurent Poirot, PhD, Juan A. Bueren, PhD, Jose C. Segovia, PhD
Journal: Blood
Blood (2016) 128 (22): 3513.
https://doi.org/10.1182/blood.V128.22.3513.3513
Published: 2016
Journal Articles

Development of Efficent Gene Therapy for the Treatment of Erythrocyte Pyruvate Kinase Deficiency.

Nestor W. Meza, Ph.D., Maria Eugenia Alonso, Susana Navarro, Ph.D, Guillermo Guenechea, Ph.D, Oscar Quintana-Bustamante, Ph.D, Maria Garcia-Gomez, Juan A. Bueren, Ph.D, Jose M. Bautista, Ph.D, Jose C. Segovia, PhD
Journal: Blood
Blood (2007) 110 (11): 2584.
https://doi.org/10.1182/blood.V110.11.2584.2584
Published: 2007
Journal Articles

Gene Therapy of the Human Erythrocyte Pyruvate Kinase Deficiency.

Jose C. Segovia, PhD, Nestor W. Meza Fellow, Oscar Quintana-Bustamante Fellow, Antonio Puyet, PhD, Paula Rio, PhD, Juan A. Bueren, PhD, Jose M. Bautista, PhD
Journal: Blood
Blood (2004) 104 (11): 1635.
https://doi.org/10.1182/blood.V104.11.1635.1635
Published: 2004
Journal Articles

Increased Generation of Hepatocytes Expressing Bone Marrow-Derived Markers after Hematopoietic Progenitors’ Mobilization in a Murine Model of Hepatic Damage.

Oscar Quintana-Bustamante Fellow, Alberto Alvarez-Barrientos, PhD, Isabel Fabregat, PhD, Juan A. Bueren, PhD, Jose C. Segovia, PhD
Journal: Blood
Blood (2004) 104 (11): 3600.
https://doi.org/10.1182/blood.V104.11.3600.3600
Published: 2004