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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

Preclinical Evaluation for the Gene Therapy of Patients with Leukocyte Adhesion Deficiency Type I

Elena Almarza, PhD, Cristina Mesa-Núñez, Carlos Damián, María Fernández-García, Begoña Díez-Cabezas, Jonathan D Schwartz, MD, Juan A Bueren
Journal: Blood
Blood (2018) 132 (Supplement 1): 5798.
https://doi.org/10.1182/blood-2018-99-111136
Published: 2018