Targeted enzyme delivery systems in lysosomal disorders: an innovative form of therapy for mucopolysaccharidosis

  • Azam Safary
  • Mostafa Akbarzadeh Khiavi
  • Yadollah OmidiEmail author
  • Mohammad A. RafiEmail author


Mucopolysaccharidoses (MPSs), which are inherited lysosomal storage disorders caused by the accumulation of undegraded glycosaminoglycans, can affect the central nervous system (CNS) and elicit cognitive and behavioral issues. Currently used enzyme replacement therapy methodologies often fail to adequately treat the manifestations of the disease in the CNS and other organs such as bone, cartilage, cornea, and heart. Targeted enzyme delivery systems (EDSs) can efficiently cross biological barriers such as blood–brain barrier and provide maximal therapeutic effects with minimal side effects, and hence, offer great clinical benefits over the currently used conventional enzyme replacement therapies. In this review, we provide comprehensive insights into MPSs and explore the clinical impacts of multimodal targeted EDSs.


Blood–brain barrier Enzyme replacement therapy Mucopolysaccharidoses Nanosystems Targeted enzyme delivery systems 



Authors would like to acknowledge the Research Center for Pharmaceutical Nanotechnology and Connective Tissue Diseases Research Center at Tabriz University of Medical Sciences for financial support. The kind help from Dr. S. Sabermoghaddam and Mrs. R. Mousavi are highly appreciated.

Compliance with ethical standards

Conflict of interest

No conflicts of interest to be disclosed.


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Copyright information

© Springer Nature Switzerland AG 2019

Authors and Affiliations

  1. 1.Connective Tissue Diseases Research CenterTabriz University of Medical SciencesTabrizIran
  2. 2.Research Center for Pharmaceutical Nanotechnology, Biomedicine InstituteTabriz University of Medical SciencesTabrizIran
  3. 3.Liver and Gastrointestinal Diseases Research CenterTabriz University of Medical SciencesTabrizIran
  4. 4.Department of Pharmaceutics, Faculty of PharmacyTabriz University of Medical SciencesTabrizIran
  5. 5.Department of Neurology, Sidney Kimmel Medical CollegeThomas Jefferson UniversityPhiladelphiaUSA

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