Rescue of a lysosomal storage disorder caused by Grn loss of function with a brain penetrant progranulin biologic.
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Logan T, et al. Among authors: przybyla l.
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
Free PMC article.