Mucopolysaccharidoses Treatment with Gene Therapy

By Daniel Walocha ‘19 Muscopolysaccharidoses (MPS) is a lysosomal storage disorder which results from a deficiency of lysosomal enzymes. The lack of enzymes causes an accumulation of glycosaminoglycans that can lead to severe symptoms including heart disease, nervous system damage, and skeletal dysplasia. Enzyme replacement therapies, hematopoietic stem cell transplantation, and substrate reduction therapy are short-lived and temporary resolutions that cannot reach a number of … Continue reading Mucopolysaccharidoses Treatment with Gene Therapy