Article

<i>Gys1</i> antisense therapy rescues neuropathological bases of murine Lafora disease

2021-02-11

Abstract excerpt

Lafora disease is a fatal progressive myoclonus epilepsy. At root, it is due to constant acquisition of branches that are too long in a subgroup of glycogen molecules, leading them to precipitate and accumulate into Lafora bodies, which drive a neuroinflammatory response and neurodegeneration. As a potential therapy, we aimed to downregulate glycogen synthase, the enzyme responsible for glycogen branch elongation,...

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Literature Corpus work: 6bc62cd2-103b-5599-8e7d-1add19982fbf
DOI: 10.1101/2021.02.11.430846

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