About Cystic Fibrosis

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:

  1. 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.
  2. 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.

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:

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.