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. Author manuscript; available in PMC: 2019 Jan 3.
Published in final edited form as: Nat Rev Neurol. 2018 Oct;14(10):606–617. doi: 10.1038/s41582-018-0057-0

Fig. 5 |. Overview of therapeutic strategies in Lafora disease.

Fig. 5 |

In addition to the viral delivery of functional laforin or malin, the therapeutic options for Lafora disease include intracellular degradation of Lafora bodies by glucan-degrading enzymes such as α-amylase, as well as the downregulation of glycogen synthesis by targeting glycogen synthase (GS) or protein phosphatase 1 (PP1) subunit R5 at the gene, RNA or protein level. ASOs, antisense oligonucleotides; RNAi, RNA interference.