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Delivery of Antisense RNA Therapeutics: Turning Negative Results Into a Positive Development

机译：递送反义RNA治疗方法：将负面结果转化为积极的发展

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摘要

The number of novel potential RNA-based antisense therapeutics is rapidly increasing. However, efficient delivery to target tissues is still the main factor that limits their translation into the clinic. Although many groups in academia and industry are working toward the development of methods to improve antisense delivery to overcome this limitation, there are very few coordinated efforts to learn from the experience of other investigators by sharing "negative" results. In the field of nucleic acid therapeutics, or any other type of therapeutics, the ultimate aim of most research projects is to develop novel or improved therapeutic strategies. It seems only logical that experiments are thought to yield a "negative result" if there is an absence of an improvement in some parameter related to potential therapeutic efficacy. These data often do not get published in scientific journals or presented at scientific meetings. However, positive and negative results obtained from scientifically sound experiments are equally valuable in facilitating progress in the field. They avoid unnecessary duplication of experiments and allow researchers to take approaches that did not yield the predicted result into account when designing new experiments.
机译：基于RNA的新型潜在反义疗法的数量正在迅速增加。然而，有效的靶组织输送仍然是限制其临床应用的主要因素。尽管学术界和工业界的许多团体都在努力开发改进反义传递的方法，以克服这一局限性，但很少有协调一致的努力通过分享“负面”结果来学习其他研究者的经验。在核酸治疗学或任何其他类型的治疗学领域，大多数研究项目的最终目标是开发新的或改进的治疗策略。如果与潜在治疗效果相关的某些参数没有改善，那么认为实验产生“负面结果”似乎是合乎逻辑的。这些数据通常不会在科学期刊上发表，也不会在科学会议上发表。然而，从科学合理的实验中获得的积极和消极结果对促进该领域的进展同样有价值。它们避免了不必要的重复实验，并允许研究人员在设计新实验时，采用不会产生预期结果的方法。

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  • 来源
    《Nucleic Acid Therapeutics》 |2021年第3期|共2页
  • 作者

    Eilers Wouter; Denti Michela A.;

  • 作者单位

    Sutura Therapeut Ltd Alderley Pk Nether Alderley SK10 4TG Cheshire England;

    Univ Trento Dept Cellular Computat &

    Integrat Biol Via Sommarive 9 I-38123 Trento Italy;

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  • 正文语种 eng
  • 中图分类 生物化学;
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