Rescue of a lysosomal storage disorder caused by Grn loss of function with a brain penetrant progranulin biologic.
Cell. 2021 Sep 2;184(18):4651-4668.e25. doi: 10.1016/j.cell.2021.08.002. Epub 2021 Aug 26.
Cell. 2021.
PMID: 34450028