Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects.

Highly significant enrichment scores were obtained for microglial and several macrophage cell populations, for both Gaa KO DBA and Gaa KO B6;129 spinal cords, with enrichment scores [-log10 (Benjamini-Hochberg p -value)] higher than 40 in all cases, suggesting the important contribution of these cell types to the spinal cord disease (Fig.

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Only a minority of disease-associated DEGs (9.5%) did not show a significant trend towards correction after AAV treatment (not rescued, Fig. 4 h).

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