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Recent progress in gene therapies for mucopolysaccharidoses

ABSTRACT Introduction: Mucopolysaccharidoses (MPSs) are lysosomal storage disorders due to deficiency of enzymes involved in glycosaminoglycan degradation. They present with multi-organ complications of variable severity and progressive neurodegeneration in most cases. Besides supportive treatments, current therapeutic options include enzyme replacement therapy and hematopoietic stem cell transplantation. However, these treatments are hampered by several risks and limitations and more effective therapies are highly needed. Areas covered: Gene therapy has been largely investigated in pre-clinical models and is now under clinical evaluation for several MPSs. This review covers recent pre-clinical data that supported ongoing or planned ex vivo and in vivo gene therapy clinical trials for MPSs. A literature search was performed using PubMed, EudraCT, ClinicalTrials.gov, and ISRCTN.com websites. Expert opinion: Gene therapy is among the most attractive new treatments for MPSs. Potential advantages of gene therapy include reduced risks and complications compared to other treatments, and improved correction of the central nervous system disorder. Nevertheless, there are major challenges to be addressed, including immune responses and possible toxicities. In upcoming years, we will learn from the ongoing trials about the most effective approaches, serotypes, routes of administration and doses to achieve the best possible results in terms of disease correction.