Gene therapies for mucopolysaccharidoses

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 levels of the therapeutic enzyme in multiple tissues either by engrafted gene-modified hematopoietic stem progenitor cells (ex vivo) or by direct infusion of a viral vector expressing the therapeutic gene (in vivo). This review focuses on the most recent clinical progress in gene therapies for MPSs. The various gene therapy approaches with their strengths and limitations are discussed.

Automated summary

Current treatments for mucopolysaccharidoses (MPSs), including enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation (HSCT), have limitations such as lack of effectiveness on brain and skeletal manifestations, lifelong injections, and high costs. Gene therapy in MPSs aims to achieve high levels of the therapeutic enzyme through gene-modified stem cells or viral vector infusion. This review discusses the recent clinical progress and various gene therapy approaches for MPSs.