Although our understanding of the disease has remarkebly improved, people with cystic fibrosis still have a significantly reduced life expectancy. Due to the hereditary disease, patients suffer from severe infections of the lungs, associated with an increasing "mucous congestion" of the respiratory tract. The reason for this is the malfunction of a chloride channel in body cells, which is caused by mutations in the gene Cystic Fibrosis Transmembrane Conductance Regulator (CFTR). Researchers have now taken a first step towards the development of a possible gene therapy for cystic fibrosis.

The research team has developed a new class of non-viral gene shuttles that can be used to transport genetic material (DNA or RNA). It is possible to specify the exact destination of the ferry, for example certain cells, using further special signal molecules. Initial tests of this gene therapy approach in the animal model were now successful: by inhaling the gene ferry it was possible to introduce a healthy copy of the CFTR gene into the genome of lung cells. The authors of the study were subsequently able to prove that the introduced gene actually produces the CFTR protein and, thus, also the chloride channel.

Under certain circumstances, the new technology could also be used for other rare lung diseases, according to the authors. First, however, further comprehensive studies are needed, which, for example, further investigate the safety of the method. If the gene is incorporated incorrectly into the genetic material, this can have serious consequences. In addition, it is not yet clear whether the method can be applied to humans.
 

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

Credit: Lungeninformationsdienst