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首页> 外文期刊>Journal of pharmaceutical sciences. >Drug delivery trends in clinical trials and translational medicine: challenges and opportunities in the delivery of nucleic acid-based therapeutics.
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Drug delivery trends in clinical trials and translational medicine: challenges and opportunities in the delivery of nucleic acid-based therapeutics.

机译：临床试验和转化医学中的药物输送趋势：基于核酸的治疗药物的输送中的挑战和机遇。

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摘要

The ability to deliver nucleic acids (e.g., plasmid DNA, antisense oligonucleotides, siRNA) offers the potential to develop potent vaccines and novel therapeutics. However, nucleic acid-based therapeutics are still in their early stages as a new category of biologics. The efficacy of nucleic acids requires that these molecules be delivered to the interior of the target cell, which greatly complicates delivery strategies and compromises efficiency. Due to the safety concerns of viral vectors, synthetic vectors such as liposomes and polymers are preferred for the delivery of nucleic acid-based therapeutics. Yet, delivery efficiencies of synthetic vectors in the clinic are still too low to obtain therapeutic levels of gene expression. In this review, we focus on some key issues in the field of nucleic acid delivery such as PEGylation, encapsulation and targeted delivery and provide some perspectives for consideration in the development of improved synthetic vectors.
机译：递送核酸（例如质粒DNA，反义寡核苷酸，siRNA）的能力提供了开发有效疫苗和新疗法的潜力。然而，基于核酸的疗法作为一种新型的生物制剂仍处于早期阶段。核酸的功效要求将这些分子递送至靶细胞内部，这极大地复杂化了递送策略并损害了效率。由于对病毒载体的安全性的考虑，优选合成载体如脂质体和聚合物用于基于核酸的治疗剂的递送。然而，临床上合成载体的递送效率仍然太低而无法获得基因表达的治疗水平。在这篇综述中，我们关注于核酸递送领域中的一些关键问题，例如聚乙二醇化，包囊和靶向递送，并为改进合成载体的开发提供了一些可供考虑的观点。

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