The Impact of Genetic Therapies for Cystic Fibrosis
Introduction
Cystic fibrosis (CF) is a genetic disorder that primarily affects the lungs and digestive system, leading to severe damage. It is caused by a mutation in the cystic fibrosis transmembrane conductance (CFTR) gene. This essay aims to provide an in-depth understanding of the impact of genetic therapies on CF.
Genetic Therapies for Cystic Fibrosis
Genetic therapies for CF aim to deliver healthy copies of the CFTR gene to the lung cells, allowing a healthy, working copy of the CFTR protein to be produced. Although the mutant copies of the CFTR gene would still be there, the presence of the correct copies would give cells the ability to make normal CFTR proteins.
Non-integrating Gene Therapy
In non-integrating gene therapy, the new copy of the CFTR gene does not disrupt the rest of the genome. This means that the risk of side effects, including cancer, is low1. However, the effect of the gene therapy might last only for several weeks or months1. A person with CF would probably need to be treated with the gene therapy repeatedly for it to be effective1.
Integrating Gene Therapy
In integrating gene therapy, a piece of DNA that contains a correct version of the CFTR gene would be delivered to an individual’s cells. The new copy of the CFTR gene would then become a permanent part of their genome. This means that a person with CF might have to receive the gene therapy only once or a few times in their life. However, there may be limited control over where the new copy of the CFTR gene integrates into the genome. This means integrating gene therapy could have undesirable side effects, such as increasing the risk of cancer.
Risks of Genetic Therapies for Cystic Fibrosis
While genetic therapies hold promise for treating CF, they also come with certain risks. One of the major risks is the body’s immune system reaction. The body may see the newly introduced viruses used in gene therapy as intruders and attack them. This can cause inflammation and, in severe cases, organ failure.
Another risk is associated with integrating gene therapy, where the new copy of the CFTR gene becomes a permanent part of the genome1. There may be limited control over where the new copy of the CFTR gene integrates into the genome1. The new copy could be inserted into a part of the genome that contains some critical information1. This means integrating gene therapy could have undesirable side effects, such as increasing the risk of cancer1.
Availability of Genetic Therapies for Cystic Fibrosis
As of now, there is no FDA-approved gene therapy for CF. However, research is ongoing, and scientists are exploring ways to provide a correct copy of the gene to treat CF. Non-integrating gene therapy has been approved by the U.S. Food and Drug Administration to treat a rare type of blindness, and it has also been shown to work in studies for hemophilia, a blood clotting disorder. In a study in England, people with CF were given a dose of a non-integrating gene therapy once per month for a year. The study indicated that the CF gene therapy was safe and resulted in a small improvement.
Statistics on Genetic Therapies for Cystic Fibrosis
To date, 27 clinical gene therapy trials involving approximately 600 CF patients have yet to achieve their desired outcomes. However, the results of a year-long trial showed only a small and variable improvement in the lung capacity of those children and adults who took part. Despite the initial enthusiasm, there is still no FDA-approved gene therapy for CF. However, the discovery of the CF gene in 1989 has paved the way for incredible progress in treating the disease such that the mean survival age of individuals living with CF is now approximately 58 years in Canada.
Conclusion
While genetic therapies for CF are not yet widely available, they hold great promise for the future. Scientists are making significant strides in understanding the risks and benefits of these therapies. As research progresses, it is hoped that more effective and safer genetic therapies will become available for individuals with CF.
References
1: Cystic Fibrosis Foundation 5: Oxford Academic
2: Journal of Translational Medicine 6: New Scientist
3: Mayo Clinic 7: Frontiers in Pharmacology
4: MDPI
