Molecular Genetics and Metabolism · 2013 · 12 citations · 45 references
MedicineGeneticsMolecular GeneticsGene DeliveryGene TherapyTransgenic Animal ModelsGenetic DisordersGenetic SyndromesGenetics Of Growth DisordersIn Vivo Gene TherapyLysosomal Storage DisordersSafety Of Gene TherapyGene Therapy For Metabolic DisordersGene Therapy In Animal ModelsGene-targeted TherapyGlycogen AccumulationGlycogen Storage DiseasesPartial ReversalPostnatal Growth Failure
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