Promising gene therapy for cystic fibrosis

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Scientists from the research group Molecular Virology and Gene Therapy of the University of Leuven have developed a new form of gene therapy that is able to cure mice with cystic fibrosis. Also, cell cultures of cystic fibrosis patients respond well to the treatment. “In the search for a cure for the disease, gene therapy should be considered again,” says the Leuven Professor Zeger Debyser.

Up to now, it has not been possible to cure the disease, which is caused by mutations in the CFTR gene. Previous research on gene therapy was unsuccessful. “As recent successes have been achieved with gene therapy for other diseases such as haemophilia and congenital blindness, we found it useful to reexamine it for cystic fibrosis,” says Debyser.

Postdoctoral researchers Dragana Vidovic and Marianne Carlon found a way to smuggle a healthy copy of the CFTR gene into the affected cells using ’recombinant adeno-associated viral vectors’ (rAAV). “The rAAV were administered to the mice via the trachea and most mice recovered from the condition. In cell cultures of tissue samples of cystic fibrosis patients, we saw a recovery of the moisture transport,” explains Debyser.

However, he warns that there are still many uncertainties and that it will take years before a therapy for patients will be available. “For example, we do not now how long the effect of the gene therapy will last,” says Debyzer.