Gene therapy gives hope

Review on the future of CF gene therapy

Professors of gene therapy, Uta Griesenbach and Eric Alton, discuss the future of CF gene therapy and briefly review fundamental therapeutic findings since the cloning of the CFTR gene two decades ago (Griesenbach & Alton, 2013). Gene therapy has the ability to correct defective genes through injecting viruses to deliver non-mutated copies of genes to the patient’s cells. Scientists believe gene therapy has the potential to cure genetic diseases in the future, thus making it an appropriate treatment for CF patients. In 2009, Griesenbach and Alton recognized that no one vector is suitable for all applications since target cells are disease-specific. Instead, the gene transfer agent (GTA) must be carefully matched to a specific target cell. In their paper, they claim that lentiviral vectors, which can insert their genomes in random locations on a patient’s chromosome, are one of the most promising developments in CF gene therapy. Research has shown that the lung is challenging to target, but the development of lentiviral vectors has been clinically proven to successfully transduce lung tissue. However, lentiviruses have the potential to impede cellular function and act as a carcinogen (Griesenbach & Alton, 2013). Therefore, it is essential for future studies to assess the safety of lentiviral vectors and continue to investigate more efficient ways to transport non-mutated genes into cells. Since CF therapeutic research is increasing rapidly, it is in my opinion that CF patients will live healthier and longer lives. 

Professor Brandon Wainwright from the CF Trust Scientific Advisory briefly discusses the mechanisms of gene therapy and how research indicates that gene therapy can create a larger extension and quality of life for CF patients. http://www.youtube.com/watch?v=ztCnd-mSsG0


Works Cited

Griesenbach, U., & Alton, E. W. (2013). Moving forward: cystic fibrosis gene therapy. Human molecular genetics, 22(R1), R52-R58.