Abstract
One of the most revolutionary technologies in recent years in the field of molecular biology is CRISPR-Cas9. CRISPR technology is a promising tool for gene editing that provides researchers the opportunity to easily alter DNA sequences and modify gene function. Its many potential applications include correcting genetic defects, treating and preventing the spread of diseases. Cystic fibrosis (CF) is one of the most common lethal genetic diseases caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. Although CF is an old acquaintance, there is still no effective/resolutive cure. Life expectancy has improved thanks to the combination of various treatments, but it is generally below average. Recently, a significant number of additional key medications have become licensed in Europe for the CF treatment including CFTR modulators. But innovative genomically-guided therapies have begun for CF and it is predictable that this will lead to rapid improvements in CF clinical disease and survival in the next decades. In this way, CRISPR-Cas9 approach may represent a valid tool to repair the CFTR mutation and hopeful results were obtained in tissue and animal models of CF disease.
Highlights
Mutations within the gene encoding for the chloride ion channel CFTR results in cystic fibrosis (CF), the most common autosomal recessive genetic disease in the Caucasian population
The Food and drug Administration (FDA) has approved a new drug, always labeled Vertex: Orkambi, a mix between ivacaftor and lumacaftor, a CFTR protein corrector that has been tested in a clinical trial on 1,108 patients over the age of 12 years homozygous F508del
The introduction of new drugs such as ivacaftor and lumacaftor improved the quality of life and increased the life expectation of Cystic fibrosis (CF) patients
Summary
One of the most revolutionary technologies in recent years in the field of molecular biology is CRISPR-Cas. A significant number of additional key medications have become licensed in Europe for the CF treatment including CFTR modulators. Innovative genomically-guided therapies have begun for CF and it is predictable that this will lead to rapid improvements in CF clinical disease and survival in the decades. In this way, CRISPR-Cas approach may represent a valid tool to repair the CFTR mutation and hopeful results were obtained in tissue and animal models of CF disease
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