Update in the Mucopolysaccharidoses
Kim L. McBride, Kevin M. Flanigan
Topics & Concepts
Enzyme replacement therapyGenetic enhancementMedicineRecombinant DNADiseaseAdeno-associated virusMultisystem diseaseGlycosaminoglycanBioinformaticsGeneInternal medicineBiologyVector (molecular biology)GeneticsAnatomyLysosomal Storage Disorders ResearchTrypanosoma species research and implicationsNeurogenetic and Muscular Disorders Research