DeepNEU: Artificially Induced Stem Cell (aiPSC) and Differentiated Skeletal Muscle Cell (aiSkMC) Simulations of Infantile Onset POMPE Disease (IOPD) for Potential Biomarker Identification and Drug Discovery.

While the undifferentiated aiPSC model with GAA deletion demonstrated a modest increase in lysosomal glycogen the differentiated aiSkMC simulations of IOPD revealed a highly significant increase in lysosomal glycogen (mean ± 99%CI = 0.585 ± 0.003) when compared with the aiSkMC WT simulations (mean ± 99%CI = −0.297 ± 0.039; Mann–Whitney u test exact p -value = 1.29E-08).

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