Disruption of the gaa Gene in Zebrafish Fails to Generate the Phenotype of Classical Pompe Disease
Wu J, Yang Y, Sun C, Sun S, Li Q, Yao Y, Fei F, Lu L, Chang Z, Zhang W, Wang X, Luo F. Disruption of the gaa Gene in Zebrafish Fails to Generate the Phenotype of Classical Pompe Disease. DNA And Cell Biology 2017, 36: 10-17. PMID: 28045567, DOI: 10.1089/dna.2016.3459.Peer-Reviewed Original ResearchConceptsGAA mRNAFrameshift mutationEnzymatic activityGene expression changesGAA geneSpecies-specific differencesPresence of autophagosomesGlycogen accumulationMutant zebrafishGAA enzymatic activityWT zebrafishZebrafish modelDays postfertilizationExpression changesZebrafishDifferent speciesPhenotypic changesAdult stageLysosomal glycogen accumulationGlycogen storage disease type IIStorage disease type IIGenesProtein levelsProtein expressionMutants