Article
Gene, RNA, and ASO-based therapeutic approaches in Cystic Fibrosis.
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Mar 2023
Allaire Normand E, Griesenbach Uta, Kerem Batsheva, Lueck John D, Stanleigh Noemie, Oren Yifat S
Abstract excerpt
Most people with Cystic Fibrosis (PwCF) harbor Cystic Fibrosis Transmembrane Conductance (CFTR) mutations that respond to highly effective CFTR modulators (HEM); however, a small fraction of non-responsive variants will require alternative approaches for treatment. Furthermore, the long-term goal to develop a cure for CF will require novel therapeutic strategies. Nucleic acid-based approaches offer the potential...
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