Nanoformulation of Therapeutic Enzymes: A Short Review

IF 16.4 1区化学 Q1 CHEMISTRY, MULTIDISCIPLINARY

Accounts of Chemical Research Pub Date : 2023-09-25 DOI:10.3311/ppch.22826

Gergő Dániel Tóth, Gábor Koplányi, Balázs Kenéz, Diána Balogh-Weiser

引用次数: 0

Abstract

Enzyme replacement therapy (ERT) is a therapeutic approach that involves the administration of specific enzymes to the patient in order to correct metabolic defects caused by enzyme deficiency. The formulation of ERTs involves the production, purification, and formulation of the enzyme into a stable and biologically active drug product, often using recombinant DNA technology. Non-systemic ERTs often involve the immobilization of the enzyme on a carrier, such as hydrogels, liposomes, or nanoparticles. ERT holds great promise for the treatment of a wide range of genetic disorders, and its success regarding lysosomal storage diseases, such as Fabry disease, Gaucher disease, and Pompe disease has paved the way for the development of similar therapies for other genetic disorders too.

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治疗酶的纳米配方:综述

酶替代疗法(ERT)是一种治疗方法，包括给患者特定的酶，以纠正由酶缺乏引起的代谢缺陷。ERTs的配方涉及酶的生产、纯化和配方，通常使用重组DNA技术，使酶成为稳定且具有生物活性的药物产品。非系统性ert通常涉及将酶固定在载体上，如水凝胶、脂质体或纳米颗粒。ERT在治疗广泛的遗传疾病方面有着巨大的希望，它在溶酶体储存疾病方面的成功，如法布里病、戈谢病和庞贝病，为其他遗传疾病的类似治疗的发展铺平了道路。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Accounts of Chemical Research 化学-化学综合

CiteScore

31.40

自引率

1.10%

发文量

312

审稿时长

2 months

期刊介绍： Accounts of Chemical Research presents short, concise and critical articles offering easy-to-read overviews of basic research and applications in all areas of chemistry and biochemistry. These short reviews focus on research from the author’s own laboratory and are designed to teach the reader about a research project. In addition, Accounts of Chemical Research publishes commentaries that give an informed opinion on a current research problem. Special Issues online are devoted to a single topic of unusual activity and significance. Accounts of Chemical Research replaces the traditional article abstract with an article "Conspectus." These entries synopsize the research affording the reader a closer look at the content and significance of an article. Through this provision of a more detailed description of the article contents, the Conspectus enhances the article's discoverability by search engines and the exposure for the research.