Advances in gene therapy for Lafora disease: Intravenous recombinant adeno-associated virus-mediated delivery of EPM2A and EPM2B genes.

A slight trend towards a decrease in NeuN‐positive cells was observed in the CA1, CA2‐CA3, and layers IV‐V of the sensorimotor cortex of Epm2a −/− and Epm2b −/− mice injected with the rAAVP31‐Null vector, though these differences were not statistically significant compared to WT mice (Figures S3A–D and S4A–D ).

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Epm2b −/− mice also displayed reduced latency to fall from the rod compared to WT controls, although this difference did not reach statistical significance.

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Moreover, we found an increasing trend in the rheobase current, although it was not statistically significant (Figure 6D ).

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Treatment with rAAVP31‐h EPM2A reduced this proportion to 42.1%, showing a clear trend towards improvement, though not reaching statistical significance (Figure 5C ).

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Treatment with rAAVP31‐h EPM2B showed a trend towards improvement (Figure 5D ).

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