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Gene Therapy For Cystic Fibrosis Shows Promise In Mice Study

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There is no known cure for cystic fibrosis (CF), but a new study shows promise that it can finally be treated through gene therapy. Researchers from the KU Leuven University in Belgium said they were able to successfully treat CF in mice and cultured human cells in their lab.

CF is a disease caused by mutations in the CFTR gene. The mutation causes chloride channels - through which water and chloride ions flow - to become dysfunctional, causing the buildup of abnormally thick secretions. The mucus can clog body parts such as the intestines, bronchi and pancreatic ducts, resulting in infection.

Some years ago, a drug that can repair dysfunctional chloride channels was developed, but the treatment only works for a few CF patients.

"Previous studies suggested that the treatment is safe, but largely ineffective for cystic fibrosis patients," said Dr. Zeger Debyser, a researcher at KU Leuven. Debyser added that because of recent successes of applying gene therapy to treat other disorders such as hemophilia and blindness, the team was encouraged to re-examine the possibility that gene therapy can also treat CF successfully.

The researchers worked on the premise that inserting healthy genes into intestinal cells can help treat the disease. For their study, they inserted copies of the normal CFTR gene into a recombinant adeno-associated virus (rAAV) genome, which then acted as a vector to deliver the gene-correcting formula to the intestine's cells. AAV was made the vector because while it can infect humans, it only elicits a light immune system response and does not cause further damage.

In gut cell cultures taken from patients, the modified AAV fixed the chloride channels. Debyser and his team were also able to note reduced mucus secretion and recovery from CF in infected mice who were given the treatment.

"We administered the rAAV to the mice via their airways. Most of the CF mice recovered. In the patient-derived cell cultures, chloride and fluid transport were restored," said Debyser.

However, the researchers also said that while they were able to succeed with the experiment, they also said that the treatment still has a long way to go before it can truly be viable as a cure for CF. Coming up with a treatment based on gene therapy may take years of work and further research that would have to involve actual CF patients.

Still, the team remains hopeful that gene therapy could become a promising form of treatment for CF patients in the future.

The findings of the study are published in the American Journal of Respiratory and Critical Care Medicine.

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