Nanotechnology applied to treatment of mucopolysaccharidoses

ABSTRACT

Introduction: Mucopolysaccharidoses (MPS) are genetic disorders caused by the accumulation of glycosaminoglycans due to deficiencies in the lysosomal enzymes responsible for their catabolism. Current treatments are not fully effective and are not available for all MPS types. Accordingly, researchers have tested novel therapies for MPS, including nanotechnology-based enzyme delivery systems and gene therapy. In this review, we aim to analyze some of the approaches involving nanotechnology as alternative treatments for MPS.

Areas covered: We analyze nanotechnology-based systems, focusing on the biomaterials, such as polymers and lipids, that comprise these nanostructures, and we have highlighted studies that describe their use as enzyme and gene delivery systems for the treatment of MPS diseases.

Expert opinion: Some protocols, such as the use of polymer-based systems or nanostructured carriers associated with enzymes and nanotechnology-based carriers for gene therapy, along with combined approaches, seem to be the future of MPS therapy.

KEYWORDS:

• Enzyme delivery system
• gene therapy
• lysosomal storage disorders
• mucopolysaccharidoses
• nanotechnology

Article highlights

• Mucopolysaccharidoses (MPS) are lysosomal disorders that lead to glycosaminoglycans (GAGs) storage.

• Among the traditional therapies as ERT and HSCT, nanotechnology-based enzyme delivery and gene therapy are potential approaches to be pursuit.

• Nonviral approaches such as nanotechnology-based carriers have shown improvements.

• Biomaterials should be chosen carefully to produce enzyme and nucleic acid nanocarriers.

This box summarizes key points contained in the article.

Declaration of interest

The authors were supported by CNPq (grant numbers 470888/2014-8 and 141742/2014-3. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed. Writing assistance from the Joshua M, American Journal Experts was utilized in the production of this manuscript and funded by CNPq.