Patricia Dickson, MD

Centennial Professor, WashU Pediatrics

Pathogenesis and therapy development for central nervous system disease due to mucopolysaccharidosis

The Dickson laboratory studies the mucopolysaccharidosis (MPS) disorders, which are lysosomal enzyme deficiencies affecting the catabolism of glycosaminoglycans. Central nervous system manifestations include progressive intellectual disability, communicating hydrocephalus, dysmyelination, spinal cord compression, and cortical atrophy. Our lab studies cerebrospinal fluid delivery of recombinant enzymes to treat central nervous disease due to MPS, and has demonstrated biodistribution of intrathecally-delivered recombinant enzymes throughout the neuroaxis of MPS models, with correction of lysosomal storage. The laboratory also studies neuroimaging and neuropathology of white matter in MPS brain and the humoral immune responses to therapeutic enzymes. Projects range from bench to bedside including clinical trials.

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