Cruz Maria Eugénia Meirinhos, Corvo Maria Luísa, Martins Maria Bárbara, Simões Sandra, Gaspar Maria Manuela
Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Av. Prof. Gama Pinto, 1649-003 Lisboa, Portugal.
Pharmaceutics. 2022 Feb 27;14(3):531. doi: 10.3390/pharmaceutics14030531.
The drugs concept has changed during the last few decades, meaning the acceptance of not only low molecular weight entities but also macromolecules as bioagent constituents of pharmaceutics. This has opened a new era for a different class of molecules, namely proteins in general and enzymes in particular. The use of enzymes as therapeutics has posed new challenges in terms of delivery and the need for appropriate carrier systems. In this review, we will focus on enzymes with therapeutic properties and their applications, listing some that reached the pharmaceutical market. Problems associated with their clinical use and nanotechnological strategies to solve some of their drawbacks (i.e., immunogenic reactions and low circulation time) will be addressed. Drug delivery systems will be discussed, with special attention being paid to liposomes, the most well-studied and suitable nanosystem for enzyme delivery in vivo. Examples of liposomal enzymatic formulations under development will be described and successful pre-clinical results of two enzymes, -Asparaginase and Superoxide dismutase, following their association with liposomes will be extensively discussed.
在过去几十年里，药物的概念发生了变化，这意味着不仅低分子量物质，而且大分子也被接受为药物的生物制剂成分。这为一类不同的分子，即一般的蛋白质，特别是酶，开启了一个新时代。酶作为治疗剂的使用在递送方面带来了新的挑战，并且需要合适的载体系统。在这篇综述中，我们将聚焦于具有治疗特性的酶及其应用，列出一些已进入医药市场的酶。还将探讨与其临床应用相关的问题以及解决其一些缺点（即免疫原性反应和循环时间短）的纳米技术策略。我们将讨论药物递送系统，特别关注脂质体，它是体内酶递送研究最充分且最合适的纳米系统。将描述正在开发的脂质体酶制剂的实例，并广泛讨论两种酶，即天冬酰胺酶和超氧化物歧化酶与脂质体结合后的成功临床前结果。
