Development of a selective antimicrobial cystic fibrosis therapy approach

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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.