Lentiviral-Mediated Gene Therapy for Severe Pyruvate Kinase Deficiency: Results from an Ongoing Global Phase 1 Study

Authors :: Ami J Shah
José Luis López Lorenzo
Julian Sevilla
Susana Navarro
Lucía Llanos
Begoña Perez de Camino Gaisse
Sol Sánchez
Josune Zubicaray
Bert Glader
May Chien
Oscar Quintana-Bustamante
Miriam Zeini
Grace Choi
Eileen Nicoletti
Gayatri Rao
Maria Grazia Roncarolo
Juan Bueren
Jonathan Schwartz
José Carlos Segovia
Source :: Transplantation and Cellular Therapy. 29:S225-S226
Publication Year :: 2023
Publisher :: Elsevier BV, 2023.

Subjects :: Transplantation
Molecular Medicine
Immunology and Allergy
Cell Biology
Hematology

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