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Codon-optimization in gene therapy: promises, prospects and challenges
Frontiers in Bioengineering and Biotechnology ( IF 5.7 ) Pub Date : 2024-03-28 , DOI: 10.3389/fbioe.2024.1371596
Anastasiia Iu Paremskaia , Anna A. Kogan , Anastasiia Murashkina , Daria A. Naumova , Anakha Satish , Ivan S. Abramov , Sofya G. Feoktistova , Olga N. Mityaeva , Andrei A. Deviatkin , Pavel Yu Volchkov

Codon optimization has evolved to enhance protein expression efficiency by exploiting the genetic code’s redundancy, allowing for multiple codon options for a single amino acid. Initially observed in E. coli, optimal codon usage correlates with high gene expression, which has propelled applications expanding from basic research to biopharmaceuticals and vaccine development. The method is especially valuable for adjusting immune responses in gene therapies and has the potenial to create tissue-specific therapies. However, challenges persist, such as the risk of unintended effects on protein function and the complexity of evaluating optimization effectiveness. Despite these issues, codon optimization is crucial in advancing gene therapeutics. This study provides a comprehensive review of the current metrics for codon-optimization, and its practical usage in research and clinical applications, in the context of gene therapy.

中文翻译:

基因治疗中的密码子优化:承诺、前景和挑战

密码子优化已经发展到通过利用遗传密码的冗余来提高蛋白质表达效率,允许单个氨基酸有多个密码子选择。最初观察到大肠杆菌,最佳密码子使用与高基因表达相关,这推动了应用从基础研究扩展到生物制药和疫苗开发。该方法对于调整基因疗法中的免疫反应特别有价值,并且具有创建组织特异性疗法的潜力。然而,挑战仍然存在,例如对蛋白质功能产生意外影响的风险以及评估优化有效性的复杂性。尽管存在这些问题,密码子优化对于推进基因治疗至关重要。这项研究全面回顾了基因治疗背景下密码子优化的当前指标及其在研究和临床应用中的实际用途。
更新日期:2024-03-28
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