Department of Physiology, BioSciences Institute, University College Cork, Ireland.
Curr Opin Pharmacol. 2022 Aug;65:102247. doi: 10.1016/j.coph.2022.102247. Epub 2022 Jun 13.
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.
本综述介绍了囊性纤维化（CF）肺部疾病中 RNA 和 DNA 为基础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传递系统，但由于所有这些策略的有效载荷都是核酸，因此应该有可能利用一项研究中的传递突破，并将这些创新应用于其他实验，以确定对每个 CF 患者的最佳策略。最终，对于每个人来说，可能是相同的方法，或者可能是针对特定突变或突变类别/组的几种不同策略。虽然目前的重点是 CF 肺部疾病，但从长远来看，目标是治疗 CF 患者所有受影响的器官，如胰腺、肠道和肝脏。
