Lentiviral-mediated Gene Therapy for Adults and Children with Severe Pyruvate Kinase Deficiency: Results from an Ongoing Global Phase 1 Study

Authors :: Ami J. Shah
José Luis López Lorenzo
Julián Sevilla
Susana Navarro
Lucía Llanos
Begoña Pérez de Camino Gaisse
Sol Sanchez
Josune Zubicaray
Bert Glader
May Chien
Oscar Quintana Bustamante
Miriam Zeini
Grace Choi
Eileen Nicoletti
Gayatri R. Rao
Maria Grazia Roncarolo
Juan A. Bueren
Jonathan D. Schwartz
José C. Segovia
Source :: Blood. 140:4902-4903
Publication Year :: 2022
Publisher :: American Society of Hematology, 2022.

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