In vivo adenine base editing of mutant Galc gene ameliorates Krabbe disease progression.

The ABE8e-treated group showed a trend toward higher base editing efficiency at the target adenine (A 6 ) in the frontal cortex and corpus callosum (0.26% ± 0.03% and 0.29% ± 0.05%, respectively) compared to the untreated (0.06% ± 0.02% in frontal cortex; 0.09% ± 0.05% in corpus callosum) and GFP-treated groups (0.05% ± 0.04% in frontal cortex; 0.04% ± 0.04% in corpus callosum) (Fig. 2 C).

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