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Paula Río, PhD
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Journal Articles

Clonal Tracking Post-Gene Therapy for Fanconi Anemia

Paula Rio, PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): SCI4.
https://doi.org/10.1182/blood-2020-133034
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

NKG2D CAR-Expressing Lymphocytes Target Acute Myeloid Leukemia Cells

Alejandra Leivas, PhD BS, MSc, Laura Córdoba, MSc, Antonio Valeri, PhD, Paula Rio, PhD, Daniel Primo, PhD, Joan Ballesteros, PhD, Lucía Fernández, PhD, Antonio Perez-Martinez, MD PhD, Dean Anthony Lee, MD PhD, Daniel J. Powell, Jr., PhD, Joaquin Martinez-Lopez, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2667.
https://doi.org/10.1182/blood-2019-123371
Published: 2019
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

NHEJ-Mediated Gene Editing, a Versatile Approach to Correct a Variety of Fanconi Anemia Genes in HSCs

Francisco J Roman-Rodriguez, PhD, Laura Ugalde, Lara A. Alvarez, Begoña Díez-Cabezas, PhD, Jordi Surrallés, PhD, Maria Jose Ramirez, PhD, Massimo Bogliolo, PhD, Helmut Hanenberg, MD PhD, Julian Sevilla, MD PhD, Sandra Rodriguez-Perales, PhD, Raul Torres-Ruiz, PhD, Juan A. Bueren, PhD, Paula Rio, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4639.
https://doi.org/10.1182/blood-2019-129946
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

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

NKG2D-CAR Transduced Primary Natural Killer Cells Efficiently Target Multiple Myeloma Cells

Alejandra Leivas, PhD BS, MSc, Paula Rio, PhD, Rebeca Mateos, MSc, Mari Liz Paciello, MD, Almudena Garcia-Ortiz, PhD, Lucia Fernandez, PhD, Antonio Perez-Martinez, MD PhD, Dean Anthony Lee, MD PhD, Daniel J. Powell, Jr., PhD, Antonio Valeri, PhD, Joaquin Martinez-Lopez, MD PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 590.
https://doi.org/10.1182/blood-2018-99-114522
Published: 2018
Journal Articles

Activated and Expanded Natural Killer Cells Expressing an NKG2D-CAR Efficiently Target Multiple Myeloma Cells

Alejandra Leivas, PhD BS, MSc, Antonio Valeri, PhD, Paula Rio, PhD, Lucia Fernandez, PhD, Iñaki Pañero, Dean Anthony Lee, MD PhD, Daniel J. Powell, Jr., PhD, Juan Jose Lahuerta, Antonio Perez-Martinez, MD PhD, Joaquin Martinez-Lopez, MD PhD
Journal: Blood
Blood (2017) 130 (Supplement 1): 4466.
https://doi.org/10.1182/blood.V130.Suppl_1.4466.4466
Published: 2017
Journal Articles

Generation Of FANCA-/- Human CD34+ Hematopoietic Stem Cells By shRNA Knockdown

Zejin Sun, MD, PhD, Rikki Enzor, Paula Rio, PhD, D. Wade Clapp, MD, Helmut Hanenberg, MD
Journal: Blood
Blood (2013) 122 (21): 2903.
https://doi.org/10.1182/blood.V122.21.2903.2903
Published: 2013
Journal Articles

Clinical Characteristics of Patients with Fanconi Anemia in Complementation Group J.

Angeles Dasi-Carpio, MD, PhD, Jose A. Casado, PhD, Guillermo Sanz, MD, Paula Rio, PhD, Maria-Teresa Santos, PhD, Juan A. Bueren, PhD, Jordi Surralles, PhD, Emilio Monteagudo, MD, Maria C. Carreras, MD
Journal: Blood
Blood (2006) 108 (11): 3769.
https://doi.org/10.1182/blood.V108.11.3769.3769
Published: 2006
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