Rescue of FTLD-associated TDP-43 pathology and neurodegeneration by peripheral AAV-mediated expression of brain-penetrant progranulin
Marvin Reich, Matthew J. Simon, Beate Polke, Georg Werner, Christian M. Schrader, Iñaki Paris, Sophie Robinson, Sonnet S. Davis, Gabrielly Lunkes de Melo, Lennart Schlaphoff, Lena Spieth, Stefan A. Berghoff, Todd Logan, Brigitte Nuscher, Katrin Buschmann, , Christian Haass
Peripheral AAV-mediated delivery of brain-penetrant PGRN rescues TDP-43 pathology, neurodegeneration and motor phenotypes in FTLD-GRN models.
bioRxiv · Jul 14, 2023 · PreprintNeuroscience