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RNA-Targeted Therapeutics

机译：RNA靶向治疗方法

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摘要

RNA-targeted therapies represent a platform for drug discovery involving chemically modified oligonucleo-tides, a wide range of cellular RNAs, and a novel target-binding motif, Watson-Crick base pairing. Numerous hurdles considered by many to be impassable have been overcome. Today, four RNA-targeted therapies are approved for commercial use for indications as diverse as Spinal Muscular Atrophy (SMA) and reduction of low-density lipoprotein cholesterol (LDL-C) and by routes of administration including subcutaneous, intravitreal, and intrathecal delivery. The technology is efficient and supports approaching "undruggable'' targets. Three additional agents are progressing through registration, and more are in clinical development, representing several chemical and structural classes. Moreover, progress in understanding the molecular mechanisms by which these drugs work has led to steadily better clinical performance and a wide range of mechanisms that may be exploited for therapeutic purposes. Here we summarize the progress, future challenges, and opportunities for this drug discovery platform.
机译：RNA靶向疗法代表了涉及化学改性的寡核蛋白的药物发现的平台，各种细胞RNA和新型靶结合基序，Watson-Crick碱基配对。克服了许多人无法思考的众多障碍被克服。如今，四个RNA针对性疗法被批准用于商业用途，以适应脊髓肌肉萎缩（SMA）和低密度脂蛋白胆固醇（LDL-C）和施用途径，包括皮下，玻璃体内和鞘内递送。该技术是高效的，支持即将到来的“不可驾拉的”目标。三个额外的代理商通过注册进展，并且在临床发展中，代表了几种化学和结构阶段。此外，了解这些药物工作的分子机制的进展情况为了稳定更好地临床表现和各种机制，可用于治疗目的。在这里，我们总结了该药物发现​​平台的进步，未来的挑战和机遇。

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