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.
| Original language | English |
|---|---|
| Pages (from-to) | 3363-3381 |
| Number of pages | 19 |
| Journal | Cellular and Molecular Life Sciences |
| Volume | 76 |
| Issue number | 17 |
| DOIs | |
| State | Published - Sep 1 2019 |
| Externally published | Yes |
Bibliographical note
Publisher Copyright:© 2019, Springer Nature Switzerland AG.
ASJC Scopus Subject Areas
- Molecular Medicine
- Molecular Biology
- Pharmacology
- Cellular and Molecular Neuroscience
- Cell Biology
Keywords
- Blood–brain barrier
- Enzyme replacement therapy
- Mucopolysaccharidoses
- Nanosystems
- Targeted enzyme delivery systems
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