Article
Chemical and genetic rescue of in vivo progranulin-deficient lysosomal and autophagic defects.
Proceedings of the National Academy of Sciences of the United States of America - 22 Jun 2021
Doyle James J, Maios Claudia, Vrancx Céline, Duhaime Sarah, Chitramuthu Babykumari, Bennett Hugh P J, Bateman Andrew, Parker J Alex
Abstract excerpt
In 2006, GRN mutations were first linked to frontotemporal dementia (FTD), the leading cause of non-Alzheimer dementias. While much research has been dedicated to understanding the genetic causes of the disease, our understanding of the mechanistic impacts of GRN deficiency has only recently begun to take shape. With no known cure or treatment available for GRN-related FTD, there is a growing need to rapidly...
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