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Journal Articles

Lentiviral-Mediated Gene Therapy for Patients with Fanconi Anemia [Group a]: Updated Results from Global RP-L102 Clinical Trials

Blood (2024) 144 (Supplement 1): 7463.

https://doi.org/10.1182/blood-2024-194638

Published: 2024

Journal Articles

CRISPR/Cas9 Multi-Editing Enhances CAR NK Cells Therapeutic Potential Against Multiple Myeloma

Blood (2024) 144 (Supplement 1): 3442.

https://doi.org/10.1182/blood-2024-207942

Published: 2024

Journal Articles

Lentiviral-mediated Gene Therapy for Patients with Fanconi Anemia [Group A]: Updated Results from Global RP-L102 Clinical Trials

Blood (2022) 140 (Supplement 1): 10646–10647.

https://doi.org/10.1182/blood-2022-168342

Published: 2022

Journal Articles

Gene Therapy for Fanconi Anemia [Group A]: Interim Results of RP-L102 Clinical Trials

Blood (2021) 138 (Supplement 1): 3968.

https://doi.org/10.1182/blood-2021-147071

Published: 2021

Journal Articles

Clonal Tracking Post-Gene Therapy for Fanconi Anemia

Paula Rio, PhD

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

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

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

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

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

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

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

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

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

Blood (2017) 130 (Supplement 1): 4466.

https://doi.org/10.1182/blood.V130.Suppl_1.4466.4466

Published: 2017

Journal Articles

Engraftment and in vivo proliferation advantage of gene-corrected mobilized CD34 + cells from Fanconi anemia patients

Paula Río, Susana Navarro, Guillermo Guenechea, Rebeca Sánchez-Domínguez, Maria Luisa Lamana, Rosa Yañez, Jose A. Casado, Parinda A. Mehta, Maria Roser Pujol, Jordi Surrallés, Sabine Charrier, Anne Galy, José C. Segovia, Cristina Díaz de Heredia, Julián Sevilla, Juan A. Bueren

Blood (2017) 130 (13): 1535–1542.

https://doi.org/10.1182/blood-2017-03-774174

Published: 2017

Includes: Supplemental data

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

Blood (2013) 122 (21): 2903.

https://doi.org/10.1182/blood.V122.21.2903.2903

Published: 2013

Journal Articles

Down-regulated expression of hsa-miR-181c in Fanconi anemia patients: implications in TNFα regulation and proliferation of hematopoietic progenitor cells

Paula Río, Xabier Agirre, Leire Garate, Rocío Baños, Lara Álvarez, Edurne San José-Enériz, Isabel Badell, José A. Casado, Marina Garín, Felipe Prósper, Juan A. Bueren

Blood (2012) 119 (13): 3042–3049.

https://doi.org/10.1182/blood-2011-01-331017

Published: 2012

Includes: Supplemental data

Journal Articles

A novel lentiviral vector targets gene transfer into human hematopoietic stem cells in marrow from patients with bone marrow failure syndrome and in vivo in humanized mice

Cecilia Frecha, Caroline Costa, Didier Nègre, Fouzia Amirache, Didier Trono, Paula Rio, Juan Bueren, François-Loïc Cosset, Els Verhoeyen

Blood (2012) 119 (5): 1139–1150.

https://doi.org/10.1182/blood-2011-04-346619

Published: 2012

Includes: Supplemental data

Journal Articles

Transforming and Tumorogenic Activity of JAK2 by Fusion to BCR: Molecular Mechanisms of Action of a Novel BCR-JAK2 Tyrosin-Kinase.

Álvaro Cuesta-Domínguez, Mara Ortega, Cristina Ormazabal, Matilde Santos-Roncero, Marta Galán-Díez, Juan Luis Steegmann, MD, PhD, Eva Arranz, María Ángeles Figuera, Jaqueline Sayyah, MS, Peter P. Sayeski, PhD, José Luis Vizmanos, Juan A. Bueren, Paula Rio, Elena Fernandez-Ruiz

Blood (2009) 114 (22): 4683.

https://doi.org/10.1182/blood.V114.22.4683.4683

Published: 2009

Journal Articles

In vivo proliferation advantage of genetically corrected hematopoietic stem cells in a mouse model of Fanconi anemia FA-D1

Paula Río, Néstor W. Meza, África González-Murillo, Susana Navarro, Lara Álvarez, Jordi Surrallés, Maria Castella, Guillermo Guenechea, José C. Segovia, Helmut Hanenberg, Juan A. Bueren

Blood (2008) 112 (13): 4853–4861.

https://doi.org/10.1182/blood-2008-05-156356

Published: 2008

NARROW

Lentiviral-Mediated Gene Therapy for Patients with Fanconi Anemia [Group a]: Updated Results from Global RP-L102 Clinical Trials

CRISPR/Cas9 Multi-Editing Enhances CAR NK Cells Therapeutic Potential Against Multiple Myeloma

Lentiviral-mediated Gene Therapy for Patients with Fanconi Anemia [Group A]: Updated Results from Global RP-L102 Clinical Trials

Gene Therapy for Fanconi Anemia [Group A]: Interim Results of RP-L102 Clinical Trials

Clonal Tracking Post-Gene Therapy for Fanconi Anemia

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

NKG2D CAR-Expressing Lymphocytes Target Acute Myeloid Leukemia Cells

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

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

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

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

Advances in the Gene Therapy of Patients with Fanconi Anemia

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

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

Engraftment and in vivo proliferation advantage of gene-corrected mobilized CD34 + cells from Fanconi anemia patients

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

Down-regulated expression of hsa-miR-181c in Fanconi anemia patients: implications in TNFα regulation and proliferation of hematopoietic progenitor cells

A novel lentiviral vector targets gene transfer into human hematopoietic stem cells in marrow from patients with bone marrow failure syndrome and in vivo in humanized mice

Transforming and Tumorogenic Activity of JAK2 by Fusion to BCR: Molecular Mechanisms of Action of a Novel BCR-JAK2 Tyrosin-Kinase.

In vivo proliferation advantage of genetically corrected hematopoietic stem cells in a mouse model of Fanconi anemia FA-D1

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American Society of Hematology

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American Society of Hematology

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