Gene Therapy For Cystic Fibrosis Yields Promising Results In Mice

Cystic fibrosis (CF)--a genetic disorder that results in buildups of mucus in the body--is caused by a mutation in the CFTR gene.

A new gene therapy now works to take a healthy copy of the CFTR gene into affected cells, according to a recent study. Researchers who presented the results at the Laboratory for Molecular Virology and Gene Therapy at KU Leuven, Belgium, found the findings very encouraging and promising for future treatment. 

This genetic disorder, also known known as mucoviscidosis, results from thick and sticky mucus that causes clogging in the body--sometimes in the airways or even the gastrointestinal tract. Fortunately, symptoms can be treated but there is no cure for the health issue. 

"A few years ago, a new drug was launched that can repair dysfunctional chloride channels", Professor Zeger Debyser said, in a news release. "Unfortunately, this medicine only works in a minority of CF patients. As for the impact of gene therapy, previous studies suggested that the treatment is safe, but largely ineffective for cystic fibrosis patients. However, as gene therapy has recently proven successful for disorders such as haemophilia and congenital blindness, we wanted to re-examine its potential for cystic fibrosis."

The improved gene therapy treatment is based on inserting the genetic material for chloride channels that are coded by the CFTR gene into the genome of a recombinant AAV viral vector, which is then derived from the innocent AAV virus, researchers say. Then, researchers used the vector bring the healthy CFTR gene copy into the affected cells. They studied this in both mice with cystic fibrosis and in gut cell cultures from CF patients--both of which yielded positive results.

"We must not give CF patients false hope. Developing a treatment based on gene therapy will take years of work. For one thing, our study did not involve actual human beings, only mice and patient-derived cell cultures. Furthermore, we still have to examine how long the therapy works. Repeated doses might be necessary. But gene therapy clearly is a promising candidate for further research towards a cure for cystic fibrosis."

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