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Antisense oligonucleotides (AOs) are an emerging therapeutic option to treat diseases with known genetic origin. AOs can be designed to target and bypass a patient’s genetic mutation. AOs have been approved by the FDA for use in Duchenne muscular dystrophy and Spinal muscular atrophy in 2016.

AOs can exclude a CF-causing mutation from the CFTR protein that will be made. This new protein could have more function than the one containing the mutation, which would then reduce the burden of disease.

This review discusses AOs and their potential use in CF and other genetic lung diseases.

 

Authors: Kelly M. Martinovich, Nicole C. Shaw, Anthony Kicic, André Schultz, Sue Fletcher, Steve D. Wilton and Stephen M. Stick

Published in Molecular and Cellular Pediatrics in February 2018.