Heather A. Lau, MD, Assistant Professor, Department of Neurology; Associate Director, Division of Neurogenetics; Director, Lysosomal Storage Disease Program at NYU Langone Health, discusses some of the treatment options for individuals with mucopolysaccharidoses (MPSs). Other than supportive treatments, current therapeutic options include enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation. However, these treatments have some limitations, and more effective therapies are highly needed.
Gene therapy is now under clinical evaluation for several MPSs and is among the most attractive new treatments for this group of rare, inherited lysosomal storage disorders that are clinically characterized by abnormalities in multiple organ systems and reduced life expectancy.
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.
