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Juan Bueren
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Journal Articles

RP-L301, a Lentiviral-Mediated Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase 2 Clinical Trial Design

Ami J Shah, José Luis López Lorenzo, Rachael F Grace, E J van Beers, M Bartels, Andreas Glenthoej, Susana Navarro, Lucia Llanos, Begoña Pérez de Camino Gaisse, Sol Sanchez, Josune Zubicaray, May Chien, Oscar Quintana Bustamante, M González-Vicent, June Iriondo, Miriam Zeini, Grace Choi, Eileen Nicoletti, Maria Chitty-Lopez, Gayatri R Rao, Juan Bueren, Jonathan D Schwartz, Jose Carlos Segovia, Julián Sevilla
Journal: Blood
Blood (2024) 144 (Supplement 1): 3583.1.
https://doi.org/10.1182/blood-2024-201734
Published: 2024
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

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
Journal: Blood
Blood (2012) 119 (5): 1139–1150.
https://doi.org/10.1182/blood-2011-04-346619
Published: 2012
Includes: Supplemental data
Journal Articles

Generation of Induced Pluripotent Stem Cells From the Peripheral Blood of a Pyruvate Kinase Decifient Patient

Oscar Quintana-Bustamante, Zita Garate, Jose Carlos Segovia, PhD, Laura Cerrato, Guillermo Güenechea, Juan Bueren
Journal: Blood
Blood (2011) 118 (21): 4713.
https://doi.org/10.1182/blood.V118.21.4713.4713
Published: 2011