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Journal Articles

Metformin for Treatment of Cytopenias in Children and Young Adults with Fanconi Anemia

Jessica A. Pollard, Elissa M. Furutani, Shanshan Liu, Erica B. Esrick, Laurie Cohen, Jacob Bledsoe, Kun Lu, Maria Jose Ramirez, Jordi Surrallés, Maggie Malsch, Ashley Kuniholm, Ashley Galvin, Myriam Armant, Annette S. Kim, Kaitlyn Ballotti, Lisa Moreau, Yu Zhou, Daria V. Babushok, Farid Boulad, Clinton Carroll, Helge Hartung, Amy Hont, Taizo A. Nakano, Timothy S. Olson, Sei-Gyung K. Sze, Alexis A Thompson, Marcin W. Wlodarski, Towia A Libermann, Alan D. D'Andrea, Markus Grompe, Edie A. Weller, Akiko Shimamura
Journal: Blood
Blood (2021) 138 (Supplement 1): 1102.
https://doi.org/10.1182/blood-2021-153598
Published: 2021
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

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

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
Journal: Blood
Blood (2017) 130 (13): 1535–1542.
https://doi.org/10.1182/blood-2017-03-774174
Published: 2017
Includes: Supplemental data
Journal Articles

Epigenetic Alterations In Fanconi Anemia: Role In Disease Progression and Therapeutic Potential

Helio Belo, Gabriela Medeiros Silva, PhD, Bruno Cardoso, PhD, Beatriz Porto, PhD, José Barbot, M.D, Jorge Coutinho, M.D, Juan A Bueren, PhD, Jordi Surralles, PhD, Jose A Casado, PhD, Manuela Benedito, M.D, Hema Saturnino, M.D, Emília Costa, M.D, Maria Gomes Silva, MD, PhD, Antonio Almeida, MD, PhD
Journal: Blood
Blood (2013) 122 (21): 4865.
https://doi.org/10.1182/blood.V122.21.4865.4865
Published: 2013
Journal Articles

On the role of FAN1 in Fanconi anemia

Brief Report
Juan P. Trujillo, Leonardo B. Mina, Roser Pujol, Massimo Bogliolo, Joris Andrieux, Muriel Holder, Beatrice Schuster, Detlev Schindler, Jordi Surrallés
Journal: Blood
Blood (2012) 120 (1): 86–89.
https://doi.org/10.1182/blood-2012-04-420604
Published: 2012
Includes: Supplemental data
Journal Articles

Origin, functional role, and clinical impact of Fanconi anemia FANCA mutations

Clinical Trials & Observations
Maria Castella, Roser Pujol, Elsa Callén, Juan P. Trujillo, José A. Casado, Hans Gille, Francis P. Lach, Arleen D. Auerbach, Detlev Schindler, Javier Benítez, Beatriz Porto, Teresa Ferro, Arturo Muñoz, Julián Sevilla, Luis Madero, Elena Cela, Cristina Beléndez, Cristina Díaz de Heredia, Teresa Olivé, José Sánchez de Toledo, Isabel Badell, Montserrat Torrent, Jesús Estella, Ángeles Dasí, Antonia Rodríguez-Villa, Pedro Gómez, José Barbot, María Tapia, Antonio Molinés, Ángela Figuera, Juan A. Bueren, Jordi Surrallés
Journal: Blood
Blood (2011) 117 (14): 3759–3769.
https://doi.org/10.1182/blood-2010-08-299917
Published: 2011
Includes: Supplemental data
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
Journal: Blood
Blood (2008) 112 (13): 4853–4861.
https://doi.org/10.1182/blood-2008-05-156356
Published: 2008
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