This review provides an update on recent developments of RNA- and DNA-based methodologies and their intracellular targets in the context of cystic fibrosis (CF) lung disease. Ultimately, clinical success will require a suitable delivery system, but since the cargo for all these strategies is nucleic acid, it should hopefully be possible to exploit delivery breakthroughs from one study and apply these innovations to other experiments in order to identify the best strategy for everyone with CF. Ultimately, it may be the same approach for everyone, or possibly a number of different strategies tailored to particular mutations or classes/groups of mutations. And whilst the current focus is on CF lung disease, in the longer term the goal is to treat all affected organs in people with CF such as the pancreas, gut, and liver.

本综述提供了在囊性纤维化 (CF) 肺病背景下基于 RNA 和 DNA 的方法及其细胞内靶点的最新进展。最终，临床成功将需要一个合适的递送系统，但由于所有这些策略的货物都是核酸，因此有望利用一项研究的递送突破并将这些创新应用于其他实验，以确定最佳策略每个人都有 CF。最终，它可能对每个人都是相同的方法，或者可能是针对特定突变或突变类别/组的许多不同策略。虽然目前的重点是 CF 肺病，但从长远来看，目标是治疗 CF 患者的所有受影响器官，例如胰腺、肠道和肝脏。