Human GLB1 knockout cerebral organoids: A model system for testing AAV9-mediated GLB1 gene therapy for reducing GM1 ganglioside storage in GM1 gangliosidosis

por Latour, Yvonne L., Yoon, Robin, Thomas, Sarah E., Grant, Christina, Li, Cuiling, Sena-Esteves, Miguel, Allende, Maria L., Proia, Richard L., Tifft, Cynthia J.
Publicado 2019