Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne’s Muscular DystrophyAngela Lek, Terence R. Flotte, Sander Pajusalu et al.|New England Journal of Medicine|2023Cited by 302
Applying genome-wide CRISPR-Cas9 screens for therapeutic discovery in facioscapulohumeral muscular dystrophyAngela Lek, Louis M. Kunkel, Andrew Kodani et al.|Science Translational Medicine|2020Cited by 79
Unexpected Death of a Duchenne Muscular Dystrophy Patient in an N-of-1 Trial of rAAV9-delivered CRISPR-transactivatorAngela Lek, Terence R. Flotte, Sander Pajusalu et al.|medRxiv|2023Cited by 34
Saturation mutagenesis-reinforced functional assays for disease-related genesKaiyue Ma, Monkol Lek, Christine O’Connor et al.|Cell|2024Cited by 18