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Mucopolysaccharidoses type I gene therapy

30 Jun 2021

Abstract excerpt

Mucopolysaccharidoses type I (MPS I) is an inherited metabolic disease characterized by a malfunction of the α-l-iduronidase (IDUA) enzyme leading to the storage of glycosaminoglycans in the lysosomes. This disease has longtime been studied as a therapeutic target for those studying gene therapy and many studies have been done using various vectors to deliver the IDUA gene for corrective treatment. Many vectors...

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PMID: 34189746
DOI: 10.1002/jimd.12414

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