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

Yvonne L. Latour(National Institute of Neurological Disorders and Stroke), Cynthia J. Tifft(Children's National), Richard L. Proia(National Institute of Diabetes and Digestive and Kidney Diseases), Miguel Sena‐Esteves(University of Massachusetts Chan Medical School), Robin Yoon(National Institutes of Health), Sarah Thomas(National Institutes of Health), Christina Grant(Children's National), Cuiling Li(National Institutes of Health), María L. Allende(National Institute of Diabetes and Digestive and Kidney Diseases)
Molecular Genetics and Metabolism Reports
September 11, 2019
10.1016/j.ymgmr.2019.100513
Cited by 58

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