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).
Bibliographical noteFunding Information:
This research was supported (in part) by grants to B.K from the Cystic Fibrosis Foundation , (grant no. KEREM15XX0 and KEREM21GO ), the Ministry of science and Technology (grant no. 3–17632 ) and the Israel Science Foundation (grant no. 2039/20 ) within the Israel Precision Medicine Partnership program and Cystic Fibrosis Foundation Research Grant ( LUECK18GO ) and NIH grant ( 1 R01 HL153988-02A1 ) to J.D.L.
This paper is part of a supplement supported by the European Cystic Fibrosis Society (ECFS).
John D. Lueck sits on the Scientific Advisory Boards for HcBioscience and The Cystic Fibrosis Foundation. Batsheva Karem is the CSO of SpliSense and Professor of Genetics in the Hebrew University, Jerusalem, Israel. Yifat is the Director of Discovery at SpliSense. This research was supported in part by Boehringer Ingelheim, Germany (BI), and BI was given the opportunity to review this presentation for medical and scientific accuracy as it relates to BI substances, as well as intellectual property considerations.
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- Cystic Fibrosis