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Delivery of Antisense RNA Therapeutics: Turning Negative Results Into a Positive Development
Nucleic Acid Therapeutics ( IF 4.0 ) Pub Date : 2021-06-04 , DOI: 10.1089/nat.2021.0028
Wouter Eilers 1 , Michela A Denti 2
Affiliation  

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.

中文翻译:

提供反义 RNA 治疗:将负面结果转化为积极发展

新型潜在的基于 RNA 的反义疗法的数量正在迅速增加。然而,有效递送至靶组织仍然是限制其转化为临床的主要因素。尽管学术界和工业界的许多团体正在努力开发改进反义传递以克服这一限制的方法,但很少有协调努力通过分享“负面”结果来学习其他研究人员的经验。在核酸治疗或任何其他类型的治疗领域,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。如果与潜在治疗功效相关的某些参数没有改善,则认为实验产生“阴性结果”似乎是合乎逻辑的。这些数据通常不会在科学期刊上发表或在科学会议上发表。然而,从科学合理的实验中获得的正面和负面结果对于促进该领域的进步同样有价值。它们避免了不必要的实验重复,并允许研究人员在设计新实验时采用未产生预测结果的方法。
更新日期:2021-06-08
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