NeuroscienceMolecular TherapyPeer-reviewed
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…·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.
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