Development of a selective antimicrobial cystic fibrosis therapy approach

囊性纤维化（CF）是欧洲最常见的遗传代谢疾病，其治疗选择不足。我们提出了一种新的治疗策略，通过直接解决导致呼吸衰竭和高CF患者死亡率的细菌感染，避免了克服人类上皮气道屏障的需要。即，选择性靶向慢性微生物肺部CF肺部感染的反义寡聚体（ASO）的可吸入应用。为了为这种新的基于ASO的CF治疗方法奠定基础，我们将设计和合成有效的和类型选择性的ASO，定义它们的生理和抗性效应，并进一步探索当配制成模块化系统时的靶向转运和释放。

Therapeutic options for Cystic Fibrosis (CF), the most common genetic metabolic disease in Europe, are insufficient. We propose a novel therapeutic strategy that obviates the need to overcome the human epithelial airway barrier by directly addressing the bacterial infections that cause respiratory failure and high CF patient mortality. Namely, an inhalable application of antisense oligomers (ASOs) targeting selectively the chronic microbial pulmonary CF lung infections. To create a basis for this new ASO-based CF therapy approach we will design and synthesize potent and type selective ASOs, define their physiological and resistance effects and further explore the targeted transport and release when formulated as modular systems.