Cystic Fibrosis Foundation Laboratory, Lexington MA, USA.
National Heart and Lung Institute, Imperial College London and the UK Respiratory Gene Therapy Consortium, UK.
J Cyst Fibros. 2023 Mar;22 Suppl 1(Suppl 1):S39-S44. doi: 10.1016/j.jcf.2022.12.016. Epub 2023 Jan 17.
Most people with Cystic Fibrosis (PwCF) harbor Cystic Fibrosis Transmembrane Conductance (CFTR) mutations that respond to highly effective CFTR modulators (HEM); however, a small fraction of non-responsive variants will require alternative approaches for treatment. Furthermore, the long-term goal to develop a cure for CF will require novel therapeutic strategies. Nucleic acid-based approaches offer the potential to address all CF-causing mutations and possibly a cure for all PwCF. In this minireview, we discuss current knowledge, recent progress, and critical questions surrounding the topic of Gene-, RNA-, and ASO-based therapies for the treatment of Cystic Fibrosis (CF).
大多数囊性纤维化（CF）患者都携带有对高效 CF 跨膜转导调节剂（HEM）有反应的囊性纤维化跨膜电导调节因子（CFTR）突变；然而，一小部分无反应的变异体将需要替代的治疗方法。此外，开发 CF 治愈方法的长期目标将需要新的治疗策略。核酸方法有可能解决所有导致 CF 的突变，并可能为所有 CF 患者提供治愈方法。在这篇简评中，我们讨论了与基因、RNA 和 ASO 治疗囊性纤维化（CF）相关的主题的当前知识、最新进展和关键问题。
