Gene, RNA, and ASO-based therapeutic approaches in Cystic Fibrosis
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society(2023)
摘要
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 to address all CF-causing mutations and possibly a cure for all PwCF. In this minireview, we discuss current knowledge, recent progress, and critical questions surrounding the topic of Gene-, RNA-, and ASO-based therapies for the treatment of Cystic Fibrosis (CF). (C) 2023 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.
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关键词
Gene,RNA,ASO,Therapeutic,Cystic Fibrosis,Delivery
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