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

by Yvonne L. Latour, Robin Yoon, Sarah E. Thomas, Christina Grant, Cuiling Li, Miguel Sena-Esteves, Maria L. Allende, Richard L. Proia, Cynthia J. Tifft
Published 2019-12-01

Cerebral organoids derived from Sandhoff disease-induced pluripotent stem cells exhibit impaired neurodifferentiation[S]

by Maria L. Allende, Emily K. Cook, Bridget C. Larman, Adrienne Nugent, Jacqueline M. Brady, Diane Golebiowski, Miguel Sena-Esteves, Cynthia J. Tifft, Richard L. Proia
Published 2018-03-01

The Ormdl genes regulate the sphingolipid synthesis pathway to ensure proper myelination and neurologic function in mice

by Benjamin A Clarke, Saurav Majumder, Hongling Zhu, Y Terry Lee, Mari Kono, Cuiling Li, Caroline Khanna, Hailey Blain, Ronit Schwartz, Vienna L Huso, Colleen Byrnes, Galina Tuymetova, Teresa M Dunn, Maria L Allende, Richard L Proia
Published 2019-12-01