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Delivering gene therapy for mucopolysaccharide diseases

12 Sept 2022

Abstract excerpt

Mucopolysaccharide diseases are a group of paediatric inherited lysosomal storage diseases that are caused by enzyme deficiencies, leading to a build-up of glycosaminoglycans (GAGs) throughout the body. Patients have severely shortened lifespans with a wide range of symptoms including inflammation, bone and joint, cardiac, respiratory and neurological disease. Current treatment approaches for MPS disorders...

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PMID: 36172050
DOI: 10.3389/fmolb.2022.965089

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