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治疗性酶:药理学改善的应用与方法

Therapeutic Enzymes: Applications and Approaches to Pharmacological Improvement.

作者信息

Yari Maryam, Ghoshoon Mohammad B, Vakili Bahareh, Ghasemi Younes

机构信息

Department of Medical Biotechnology, School of Advanced Medical Sciences and Technologies, Shiraz University of Medical Sciences, Shiraz. Iran.

Pharmaceutical Sciences Research Center, Shiraz University of Medical Sciences, Shiraz. Iran.

出版信息

Curr Pharm Biotechnol. 2017;18(7):531-540. doi: 10.2174/1389201018666170808150742.

DOI:10.2174/1389201018666170808150742
PMID:28786356
Abstract

BACKGROUND

Among therapeutic proteins, enzymes represent small and of course profitable market. They can be used to treat important, rare, and deadly diseases. Enzyme therapy is the only available treatment for certain disorders.

METHOD

Here, pharmaceutical enzymes are reviewed. They are categorized in four main groups, enzymes in replacement therapy, enzymes in cancer treatment, enzymes for fibrinolysis, and finally enzymes that are used topically for various treatments. Furthermore, enzyme gene therapy and future perspective of therapeutic enzymes are mentioned in brief.

RESULTS

There are many important approved enzymes in pharmaceutical market. Several approaches such as point mutation, fusion protein designing, glycoengineering, and PEGylation were used to achieve improved enzymes. Although sometimes enzymes were engineered to facilitate production and purification process, appropriate delivery to target sites, extending half-life, and reducing immunogenicity are among the main goals of engineering approaches.

CONCLUSION

Overall, enzymes play a critical role in treatment of common and rare diseases. Evaluation of new enzymes as well as improvement of approved enzymes are of the most important challenges in biotechnology.

摘要

背景

在治疗性蛋白质中,酶代表着一个规模虽小但利润可观的市场。它们可用于治疗重要、罕见和致命疾病。酶疗法是某些疾病唯一可用的治疗方法。

方法

在此,对药物用酶进行综述。它们被分为四大类,即替代疗法中的酶、癌症治疗中的酶、纤维蛋白溶解用酶,以及最后用于各种局部治疗的酶。此外,还简要提及了酶基因疗法和治疗性酶的未来展望。

结果

在药品市场上有许多重要的已获批酶。人们采用了多种方法,如点突变、融合蛋白设计、糖基工程和聚乙二醇化,以获得改良的酶。尽管有时对酶进行工程改造是为了便于生产和纯化过程,但将其适当递送至靶位点、延长半衰期和降低免疫原性是工程方法的主要目标。

结论

总体而言,酶在常见和罕见疾病的治疗中发挥着关键作用。评估新酶以及改进已获批的酶是生物技术领域最重要的挑战之一。

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