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Setting Back the Clock: Adenoviral-Mediated Gene Therapy for Lysosomal Storage Disorders
Lysosomal storage diseases (LSD) arise from mutations in the genes for lysosomal proteins that degrade and recycle macromolecules (Futerman and van Meer, 2004; Mach, 2002; Vellodi, 2005; Walkley, 2001). The un...