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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