Articles with "penetrant pgrn" as a keyword



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PGRN deficiency exacerbates, whereas a brain penetrant PGRN derivative protects, GBA1 mutation-associated pathologies and diseases

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Published in 2022 at "Proceedings of the National Academy of Sciences of the United States of America"

DOI: 10.1073/pnas.2210442120

Abstract: Significance This study carries immediate significance and novelty from following perspectives: (a) It provides the first line of in vivo and ex vivo evidence demonstrating the crucial role of PGRN in GBA1 mutation-related diseases (i.e.,… read more here.

Keywords: penetrant pgrn; gba1 mutation; brain penetrant; pgrn ... See more keywords