Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: A Global Phase 1 Study for Adult and Pediatric Patients Lorenzo, J., Navarro, S., Shah, A. J., Roncarolo, M., Sevilla, J., Llanos, L., de Gaisse, B., Sanchez, S., Glader, B., Chien, M., Bustamante, O., Beard, B. C., Law, K. M., Zeini, M., Choi, G., Nicoletti, E., Bueren, J. A., Rao, G. R., Schwartz, J. D., Segovia, J. C. AMER SOC HEMATOLOGY. 2020

View details for DOI 10.1182/blood-2020-137246

View details for Web of Science ID 000607547204296