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Fluids and Barriers of the CNS|April 11, 2024
C9ORF72 patient-derived endothelial cells drive blood-brain barrier disruption and contribute to neurotoxicityAna Aragón-González, Allan C Shaw, Jannigje R Kok, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|July 23, 2023
Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primatesSamantha Powers, Shibi Likhite, Kamal K Gadalla, et al.Cell Reports|December 8, 2022
Mechanisms of IRF2BPL-related disorders and identification of a potential therapeutic strategyShrestha Sinha Ray, Debdeep Dutta, Cassandra Dennys, et al.Neurotherapeutics : the Journal of the American Society for Experimental Neurotherapeutics|June 14, 2024
MeCP2 gene therapy ameliorates disease phenotype in mouse model for Pitt Hopkins syndromeCassandra N Dennys, Sheryl Anne D Vermudez, Robert J M Deacon, et al.Science Advances|January 19, 2022
Loss of IRF2BPL impairs neuronal maintenance through excess Wnt signalingPaul C Marcogliese, Debdeep Dutta, Shrestha Sinha Ray, et al.Nature Communications|July 4, 2025
Targeting microRNA-dependent control of X chromosome inactivation improves the Rett Syndrome phenotypeSong Lou, Rachisan DJiake Tihagam, Urszula N Wasko, et al.Pageof 2