New Gene-Delivery Systems Raise Hope for Patients with Cystic Fibrosis

News 17-2019 EN

Although research is advancing with great strides, the life expectancy of people with cystic fibrosis (CF) is still significantly reduced. The genetic disorder causes severe infections of the lungs associated with the increasing accumulation of mucus clogging the airways.

CF is caused by mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene, resulting in the dysfunction of a chloride channel in somatic cells. Researchers have now succeeded in taking an initial step toward the development of a potential gene therapy for cystic fibrosis.

The research team has developed a new class of non-viral gene-delivery systems, inside which genetic material (DNA or RNA) can be packaged. Through further specific signaling molecules, it is also possible to specify the precise destination of the delivery system, such as certain cells. Initial tests of this gene-therapy approach in an animal model have been successful: By inhalation of the delivery system, the researchers managed to introduce a healthy copy of the CFTR gene into the genome of lung cells. The authors of the study were then also able to prove that the introduced gene actually produced the CFTR protein as well as the chloride channel.

According to the authors of the study, this new technology may also be used for other rare lung diseases. First of all, however, further comprehensive studies are necessary to evaluate various factors, such as the safety of the method. Because if the gene is introduced incorrectly into the genome, this may have serious consequences. Moreover, it is not yet clear whether the method can be extrapolated to humans.

Further Information

Original publication:

Shan Guan et al.: Self-assembled peptide – poloxamine nanoparticles enable in vitro and in vivo genome restoration for cystic fibrosis. In: Nature Nanotechnology 2019; doi: 10.1038/s41565-018-0358-x

Source: Lung Information Service