Schuh Roselena S, Baldo Guilherme, Teixeira Helder F
a Programa de Pós-Graduação em Ciências Farmacêuticas da UFRGS , Faculdade de Farmácia , Porto Alegre , RS , Brazil.
b Programa de Pós-Graduação em Genética e Biologia Molecular da UFRGS , Departamento de Fisiologia , Porto Alegre , RS , Brazil.
Expert Opin Drug Deliv. 2016 Dec;13(12):1709-1718. doi: 10.1080/17425247.2016.1202235. Epub 2016 Jun 30.
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.
黏多糖贮积症(MPS)是由于负责糖胺聚糖分解代谢的溶酶体酶缺乏,导致糖胺聚糖蓄积而引起的遗传性疾病。目前的治疗方法并不完全有效,且并非适用于所有类型的MPS。因此,研究人员已经对MPS的新型疗法进行了测试,包括基于纳米技术的酶递送系统和基因疗法。在本综述中,我们旨在分析一些涉及纳米技术作为MPS替代治疗方法的途径。涵盖领域:我们分析基于纳米技术的系统,重点关注构成这些纳米结构的生物材料,如聚合物和脂质,并着重介绍了将其用作治疗MPS疾病的酶和基因递送系统的研究。专家观点:一些方案,如使用基于聚合物的系统或与酶相关的纳米结构载体以及基于纳米技术的基因治疗载体,再加上联合方法,似乎是MPS治疗的未来方向。