Cystic fibrosis is the most common genetic disease in the world. More than 70.000 people around the globe are affected. The disease manifests itself by letting a thick layer of mucus appear in the lungs, which provides a bacteria-friendly environment. The disease is fatal for sufferers, who tend to die very young. The average age of death is 41 in the US and the basic management of the disease requires constant use of antibiotics and physiotherapy.
Scientists have identified the gene responsible for cystic fibrosis 26 years ago. Since then, teams of researchers have gathered their efforts to use all their knowledge and offer help in a future and efficient treatment for the illness.
After a history of study, research, analysis and development, doctors have discovered that gene therapy can help sufferers who confront themselves with the devastating consequences of the affection.
The findings come from a recent study performed on 136 patients who received monthly doses of gene therapy in some cases and placebo in other cases, to compare, contrast and strengthen the conclusions. It seems that patients who were given the gene therapy have evolved to a slight but progressive level of healing of lung functions, compared to those who received a placebo only.
The treatment consists of vapors containing fatty bubbles, also known as liposomes. Patients are treated as they inhale the compound. The treatment is laced with healthy DNA that managed to boost the immunity of treated patients.
After experimenting the new treatment on 62 patients, researchers observed improvement in the lung functions of all study subjects. Their health status was regularly tested by measuring the quantity of air they were capable to exhale in one second. Over the course of a year, those who benefited from the treatment exhibited 3.7% better lung function compared to those who were given placebo medication only.
The improvement is not as high as expected, but future analysis and development of the gene treatment is expected to show much better results. Experts comment that the difference supported by the drug “could mean the difference between needing a lung transplant at the age of 60 instead of 40.”
Perfect doses must be further determined and more advanced studies must be performed. The future is still uncertain, as giant investments must be made to improve the treatments providing better and more efficient alternatives. Gene therapy attacks the basic flaw of cystic fibrosis and owns the potential to reduce the classic and low-in-performance routine of medication for sufferers. Gene therapy can help in cystic fibrosis treatments to a large extent, but provided large budgets are invested on a more relevant, large scale study, somewhere in the future.
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