Purpose

Advancements in genetic engineering have profoundly impacted the biotherapeutic industry and shaped many of the methods and strategies used today for both bioproduction and therapeutic design. Improvements in production yield and quality have been achieved by manipulating the expression of genes involved in cell survival, metabolism, secretion, and glycosylation, among others, and recent breakthroughs in gene therapy clinical trials show great promise for the future. Here we review the current genome editing tools used in the field and examine major areas of research for microbial and mammalian host bioproduction as well as progress in gene and cell-based therapeutics.

Methods

We summarized gene editing methods used for both prokaryote and eukaryote-based bioproduction and provide a brief survey of clinical approaches for gene therapy.

Results

While early advances in genetic engineering have create ideal prokaryotic host cells for bioproduction, the advent of programmable nucleases has greatly expanded the utility of eukaryotic hosts for glycoprotein production, enabling more precise control over these cell factories. These same tools have also created new opportunities for the creation of gene therapy-based therapeutics with several clinical trials now underway.

Conclusion

Genetic engineering has changed the biotherapeutic landscape and created novel avenues for bioproduction and therapeutic design.

中文翻译：

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基因工程在生物治疗学发展中的应用

目的

基因工程的进步深刻地影响了生物治疗行业，并影响了当今用于生物生产和治疗设计的许多方法和策略。通过操纵涉及细胞存活，代谢，分泌和糖基化等基因的表达，已经实现了产量和质量的提高，并且基因治疗临床试验中的最新突破显示了未来的广阔前景。在这里，我们回顾了当前在该领域中使用的基因组编辑工具，并考察了微生物和哺乳动物宿主生物生产以及基因和基于细胞的疗法方面的主要研究领域。

方法

我们总结了用于原核生物和基于真核生物的生物生产的基因编辑方法，并对基因治疗的临床方法进行了简要概述。

结果

尽管基因工程的早期进展为生物生产创造了理想的原核宿主细胞，但可编程核酸酶的出现极大地扩大了真核宿主用于糖蛋白生产的效用，从而能够更精确地控制这些细胞工厂。这些相同的工具也为基于基因疗法的疗法创造了新的机会，目前正在进行多项临床试验。

结论

基因工程改变了生物治疗的格局，并为生物生产和治疗设计创造了新的途径。