Third party funded individual grant
Acronym: FI899/11-1
Start date : 01.09.2023
End date : 31.08.2026
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.
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.