Peripheral Expression of Brain-Penetrant Progranulin Rescues Pathologies in Mouse Models of Frontotemporal Lobar Degeneration

Researchers developed an adeno-associated virus targeting the liver to achieve sustained peripheral expression of a transferrin receptor binding, brain-penetrant PGRN variant in two mouse models of FTLD-GRN, namely, Grn knockout and GrnxTmem106b double knockout mice.
[Science Translational Medicine]
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