Since the discovery of the CFTR gene, researchers have been working to develop a gene therapy technique that helps to correct the causative gene in cystic fibrosis patients. Many vector delivery systems have been researched. However, even after years of clinical trials, there is still no FDA approved cystic fibrosis gene therapy technique. Researchers have found difficulty finding a vector system that has maintained high levels of expression over an extended period of time. This review investigates the early and current development of vector delivery systems, including viral and non-viral vectors, in a comparative study measuring efficiency. Currently, lentivirus vectors are a promising viral technique for CF patients, as well as nanoparticle delivery systems due to their enhanced gene expression and delivery efficiency. While the implementation of a cystic fibrosis gene therapy technique has been more challenging than previously expected, new research continues to bring the vector systems closer to reality.
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Barwatt, H. (2020). Cystic Fibrosis Gene Therapy: Vector Systems. D.U.Quark, 4 (2). Retrieved from https://dsc.duq.edu/duquark/vol4/iss2/2