Device Delivers Gene Therapy for Lung Disease

Drug-delivery researchers have developed a device with the potential to improve gene therapy for patients with inherited lung diseases such as cystic fibrosis. In cell culture and mouse models, scientists demonstrated a novel technique for the aerosolization of inhalable nanoparticles that can be used to carry messenger RNA, the technology underpinning COVID-19 vaccines, to patients’ lungs.

The findings are important because the current nebulization method for nanoparticles subjects them to shear stress, hindering their ability to encapsulate the genetic material and causing them to aggregate in certain areas of the lungs rather than spread out evenly, the researchers said.

One faulty gene — the cystic fibrosis transmembrane conductance regulator, or CFTR — causes the disease, which is characterized by lung dehydration and mucus buildup that blocks the airway.

With the coronavirus vaccines, the mRNA carried by the lipid nanoparticles instructs cells to make a harmless piece of the virus’ spike protein, which triggers an immune response from the body. As a therapy for cystic fibrosis, the genetic material would fix the flaw in patients’ CFTR gene.

The team utilized a novel microfluidic chip that helps in generation of plumes that carry nanoparticles and does not cause any shear stress. The device is based on the similar idea of an inkjet cartridge that generates plumes to print words on paper. (Image Credit: Oregon State University)

For more information, visit here .