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Lentiviral-mediated gene therapy for severe pyruvate kinase deficiency: results from an ongoing global phase 1 study

Jose Luis Lopez Lorenzo,Ami Shah,Julian Sevilla,Susana Navarro,Lucia Llanos,Begona Perez de Camino Gaisse, Sol Sanchez,Josune Zubicaray,Bertil Glader,May Chien,Oscar Quintana Bustamante,Miriam Zeini,Grace Choi,Eileen Nicoletti,Gayatri Rao,Maria Grazia Roncarolo,Juan Bueren,Jonathan Schwartz,Jose Carlos Segovia

Transplantation and Cellular Therapy（2023）

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severe pyruvate kinase deficiency,pyruvate kinase deficiency,pyruvate kinase,gene therapy,lentiviral-mediated
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