Lentiviral-mediated Gene Therapy for Adults and Children with Severe Pyruvate Kinase Deficiency: Results from an Ongoing Global Phase 1 Study
- Resource Type
- Abstract
- Authors
- Shah, Ami J.; López Lorenzo, José Luis ; Sevilla, Julián ; Navarro, Susana ; Llanos, Lucía ; Pérez de Camino Gaisse, Begoña ; Sanchez, Sol ; Zubicaray, Josune ; Glader, Bert; Chien, May; Quintana Bustamante, Oscar ; Zeini, Miriam ; Choi, Grace ; Nicoletti, Eileen ; Rao, Gayatri R. ; Roncarolo, Maria Grazia; Bueren, Juan A. ; Schwartz, Jonathan D.; Segovia, José C.
- Source
- In Blood 15 November 2022 140 Supplement 1:4902-4903
- Subject
- Language
- ISSN
- 0006-4971