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

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.