century, advances in clinical management have reduced disease burden and increased longevity. These advances are amplified by the recent development of highly effective CFTR modulator therapies (HEMT) that provided remarkable clinical results in people with CF (pwCF). However, some pwCF do not benefit from HEMT due to several limiting factors. Genetic therapies have increasingly emerged as prospective, complementary treatments to HEMTs for those unable to benefit from HEMT. Genetic therapies have yet to be approved for clinical use in pwCF, but multiple clinical trials are in progress. In anticipation of future approval of one or more of these candidates, it is essential to identify and discuss how these genetic therapies might be used in early life, when many CF lesions originate, especially for those with severe mutations. In this multidisciplinary review, we discuss several pertinent factors such as CFTR localization and function, CF disease origins, emerging developments in genetic therapies, ethical considerations and other perspectives needed to guide future genetic therapies in early life.
Cooney et al. (Wed,) studied this question.