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Development of novel therapeutics for all individuals with CF (the future goes on)
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Accepted Version
Date
2022-10-30
Authors
Amaral, Margarida D.
Harrison, Patrick T.
Journal Title
Journal ISSN
Volume Title
Publisher
Elsevier B.V.
Published Version
Abstract
Despite the major advances and successes in finding and establishing new treatments that tackle the basic defect in Cystic Fibrosis (CF), there is still an unmet need to bring these potentially curative therapies to all individuals with CF. Here, we review aspects of what is still missing to treat all individuals with CF by such approaches. On the one hand, we discuss novel holistic (high-throughput) approaches to elucidate mechanistic defects caused by distinct classes of mutations to identify novel drug targets. On the other hand, we examine therapeutic approaches to correct the gene in its own environment, i.e., in the genome.
Description
Keywords
CFTR , Cystic fibrosis , Genome editing , Theratypes
Citation
Amaral, M. D. and Harrison, P. T. (2022) 'Development of novel therapeutics for all individuals with CF (the future goes on)', Journal of Cystic Fibrosis. doi: 10.1016/j.jcf.2022.10.007