Targeting GLB1 in mice by CRISPR/Cas9 genome editing: Establishing a novel model for type II GM1 gangliosidosis
Nicoli E-R., Han T., Yoon R., Glase B., Latour Y., Huebecker M., D'Souza A., Peiravi M., Tyrlik M., Springer D., Jeeva JM., Donahue D., Sidhu R., Kell P., Jiang X., Acosta M., Platt FM., Proia RL., Tifft CJ.