Litcius/Paper detail

Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne’s Muscular Dystrophy

Angela Lek

2023New England Journal of Medicine11 citationsDOI

Topics & Concepts

MedicineDuchenne muscular dystrophyMuscular dystrophyGenetic enhancementPediatricsGeneInternal medicineGeneticsBiologyVirus-based gene therapy researchCAR-T cell therapy researchCRISPR and Genetic Engineering