Gene Therapy for Adult and Pediatric Patients with Severe Pyruvate Kinase Deficiency: Results from a Global Study of RP-L301
Ami Shah,Julian Sevilla,Jose Luis Lopez Lorenzo,Susana Navarro, Lucia Llanos, Belgona Perez de Camino Gaisse, Sol Sanchez,Josune Zubicaray, Bert Glader,May Chien,Oscar Quintana Bustamante,Marta Gonzalez Vicent,June Iriondo,Michael Rothe,Philipp John-Neek, Antonella Bastone,Axel Schambach,Miriam Zeini, Grace Choi,Eileen Nicoletti,Gayatri Rao,Maria-Grazia Roncarolo,Juan A. Bueren,Jonathan Schwartz,Jose-Carlos Segovia MOLECULAR THERAPY（2024）
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