Cystic Fibrosis gene therapy: possible cure?
In 1989, experts discovered the gene that causes cystic fibrosis and identified it as the cystic fibrosis transmembrane conductance regulator or CFTR. The discovery of this defective gene posed new possibilities of a cure.
Gene Therapy
Experts proposed gene therapy as a plausible method for curing the disease. Gene Therapy is the process of creating a healthy version of the flawed CFTR gene and infusing it into the affected cells in the body, particularly into the lungs of cystic fibrosis-inflicted patients. Said process defies convention, since it doesn’t just treat the disease symptoms; it corrects the illness.
Methods of Gene Transfer in Gene Therapy
The gene is transferred into a cell through an agent. There are two types of agents:
- Viruses
Through gene therapy, scientists have utilized the way a virus penetrates into the body’s cells. How do scientists control this? Scientists replace the viral genes with the healthy gene of a virus and enable the virus to enter the cell. - Liposomes
These substances are composed of fat that latch on to cells. Scientists came up with the idea to attach the healthy genetic material to the liposomes so that the liposomes carry it as they enter the cell.
Gene therapy evolution
In the quest for the cure of cystic fibrosis, it was proven effective to prevent the CFTR from further damaging the cells of the patient, thanks to experiments done in creating a healthy copy of the defective gene. Initial experiments were made on animals.
- Adenoviral vector
Scientists infused the adenovirus with an object called the adenoviral vector through the nose and mouth. Scientists then experimented on humans, but experiments were stopped when the human body reacted negatively to the infusion. The lungs showed signs of inflammation. Added to this, the cure seemed to be only temporary. - Adeno-associated virus-based vector
Afterwards, a safer method was developed to inject the virus. Experts decided to make use of an adeno-associated virus-based vector, which generally did not show signs of affecting other parts of the body. The second method was proven safe in clinical tests, but did not really result in any improvement in the lungs. So as not to risk the safety of test subjects, a consensus was reached to halt experiments. - Alternative Methods to The Viral Vector
Due to the imperfection of gene therapy, researchers tried using non-viral means to infuse the healthy gene. The biggest advantages of a non-viral gene transfer agent lies in the fact that they are non-infectious in comparison to viruses and can be produced easier on a large scale. On the other hand, they have a lower gene transfer capability than viruses.
Drawbacks to the cure of Cystic Fibrosis
None of the above methods have been able to have effect for more than a couple of days. Despite the drawbacks, gene therapy has been considered the only known process of curing cystic fibrosis. Gene therapy, though, has yet to prove itself as truly effective in curing the disease.
The Quest Continues
Although the methods mentioned did not result in a successful cure to cystic fibrosis, scientists remain positive about gene therapy. There are 2 main areas that need be improved in the future for better results:
- A more guaranteed method of infusing the healthy gene into the affected cells.
- Finding a way to make the cure permanent.
Scientists are already working on these problems and will continue clinical trials with new and improved gene therapy agents with longer effects in cystic fibrosis patients.
Disclaimer: This website provides general information about cystic fibrosis and in no means should be taken as a medical or health advice. Please consult your doctor before acting on any of its information.