爱魔域

小核酸通过一系列过程调节基因表达的能力已被广泛探索。与传统治疗相比，机战久久私服组件升星怎么最快小核酸治疗有可能通过基因编辑实现持久甚至疗效。由于最近的技术进步，已经实现了用于治疗和生物医学应用的高效小核酸递送，加速了其临床转化。在这里，我们回顾了越来越多的小核酸治疗类别以及最常见的化学修饰和递送平台。我们还讨论了每个递送平台的设计、开发和治疗应用方面的关键进展。此外，本综述还全面介绍了目前已批准的小核酸药物，包括 11 种反义寡核苷酸 （ASO）、2 种适配体和 6 种 siRNA 药物，总结了它们的修饰、疾病特异性作用机制和递送策略。还讨论了临床试验状态已被记录和更新的其他候选者。我们还考虑了战略问题，例如重要的安全考虑、新的媒介和将学术突破转化为临床的障碍。小核酸治疗药物在临床试验中产生了良好的结果，并有可能解决以前“不可成药”的靶点，这表明它们可能有助于指导其他临床候选药物的开发。

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Landscape of small nucleic acid therapeutics: moving from the bench to the clinic as next-generation medicines

The ability of small nucleic acids to modulate gene expression via a range of processes has been widely explored. Compared with conventional treatments, small nucleic acid therapeutics have the potential to achieve long-lasting or even curative effects via gene editing. As a result of recent technological advances, efficient small nucleic acid delivery for therapeutic and biomedical applications has been achieved, accelerating their clinical translation. Here, we review the increasing number of small nucleic acid therapeutic classes and the most common chemical modifications and delivery platforms. We also discuss the key advances in the design, development and therapeutic application of each delivery platform. Furthermore, this review presents comprehensive profiles of currently approved small nucleic acid drugs, including 11 antisense oligonucleotides (ASOs), 2 aptamers and 6 siRNA drugs, summarizing their modifications, disease-specific mechanisms of action and delivery strategies. Other candidates whose clinical trial status has been recorded and updated are also discussed. We also consider strategic issues such as important safety considerations, novel vectors and hurdles for translating academic breakthroughs to the clinic. Small nucleic acid therapeutics have produced favorable results in clinical trials and have the potential to address previously “undruggable” targets, suggesting that they could be useful for guiding the development of additional clinical candidates.