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

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Abstract

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.

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Fig. 1
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Data were adapted with permission from a work published by Baldo et al. [101]

Fig. 4

Data were adapted with permission from a work published by Sonoda et al. [108]

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Acknowledgements

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.

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  1. Azam Safary and Mostafa Akbarzadeh Khiavi have an equal contribution as the joint first authors.

Authors and Affiliations

  1. Connective Tissue Diseases Research Center, Tabriz University of Medical Sciences, Tabriz, Iran

    Azam Safary

  2. Research Center for Pharmaceutical Nanotechnology, Biomedicine Institute, Tabriz University of Medical Sciences, Tabriz, 51656-65811, Iran

    Azam Safary, Mostafa Akbarzadeh Khiavi & Yadollah Omidi

  3. Liver and Gastrointestinal Diseases Research Center, Tabriz University of Medical Sciences, Tabriz, Iran

    Mostafa Akbarzadeh Khiavi

  4. Department of Pharmaceutics, Faculty of Pharmacy, Tabriz University of Medical Sciences, Tabriz, Iran

    Yadollah Omidi

  5. Department of Neurology, Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, PA, 19107, USA

    Mohammad A. Rafi

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  1. Azam Safary
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Safary, A., Akbarzadeh Khiavi, M., Omidi, Y. et al. Targeted enzyme delivery systems in lysosomal disorders: an innovative form of therapy for mucopolysaccharidosis. Cell. Mol. Life Sci. 76, 3363–3381 (2019). https://doi.org/10.1007/s00018-019-03135-z

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