AAV-mediated progranulin delivery to a mouse model of progranulin deficiency causes T cell-mediated hippocampal degeneration
Defne A. Amado, Julianne Rieders, Fortunay H. Diatta, Pilar Hernandez‐Con, Adina Singer, Jordan T. Mak, Junxian Zhang, Eric Lancaster, Beverly L. Davidson, Alice S. Chen‐Plotkin
This study shows that AAV-mediated GRN overexpression in mice causes hippocampal degeneration and T cell infiltration, raising safety concerns for GRN-based CNS gene therapies.
Molecular Therapy · Nov 16, 2018Peer-reviewedNeuroscience