机译:慢病毒造血干细胞基因疗法治疗早发性异染性脑白质营养不良:非随机、开放标签、1/2 期试验和扩大可及性的长期结果
Unit Neurol,IRCCS San Raffaele Sci Inst;
San Raffaele Telethon Inst Gene Therapy SR Tiget,IRCCS San Raffaele Sci Inst;
Dept Chem Biol & Biotechnol,Univ PerugiaUniv Ctr Stat Biomed Sci CUSSBUnit Hematol & Bone Marrow Transplantat,IRCCS San Raffaele Sci InstNeuroradiol Unit,IRCCS San Raffaele Sci InstPediat Immunohematol Unit,IRCCS San Raffaele Sci InstClin Mol Biol Lab,IRCCS San Raffaele Sci InstDept Anesthesia & Crit Care,IRCCS San Raffaele Sci InstOrchardTherapeut Europe;
CORD BLOOD TRANSPLANTATION; RECONSTITUTION; ABNORMALITIES; OUTCOMES; CHILDREN;