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Gene therapies for mucopolysaccharidoses

19 May 2023

Abstract excerpt

Current specific treatments for mucopolysaccharidoses (MPSs) include enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation (HSCT). Both treatments are hampered by several limitations, including lack of efficacy on brain and skeletal manifestations, need for lifelong injections, and high costs. Therefore, more effective treatments are needed. Gene therapy in MPSs is aimed at obtaining high...

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PMID: 37204267
DOI: 10.1002/jimd.12626

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