Nanotechnology applied to treatment of mucopolysaccharidoses.
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.
a Programa de Pós-Graduação em Ciências Farmacêuticas da UFRGS , Faculdade de Farmácia , Porto Alegre , RS , Brazil.
Roselena S
RS
Schuh
b Programa de Pós-Graduação em Genética e Biologia Molecular da UFRGS , Departamento de Fisiologia , Porto Alegre , RS , Brazil.
Guilherme
G
Baldo
a Programa de Pós-Graduação em Ciências Farmacêuticas da UFRGS , Faculdade de Farmácia , Porto Alegre , RS , Brazil.
Helder F
HF
Teixeira
D056947:Enzyme Replacement Therapy / Q000379:methods; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D006025:Glycosaminoglycans / Q000378:metabolism; D006801:Humans; D009083:Mucopolysaccharidoses / Q000628:therapy*; D049329:Nanostructures; D036103:Nanotechnology / Q000379:methods*
D006025:Glycosaminoglycans
Enzyme delivery system; gene therapy; lysosomal storage disorders; mucopolysaccharidoses; nanotechnology