Potential inhalable form of gene therapy for lung diseases developed

Researchers at the Koch Institute for Integrative Cancer Research and Institute for Medical Engineering and Science, have developed an inhalable form of messenger RNA that has the potential to be administered straight to the lungs to treat cystic fibrosis and other lung diseases.
In genetic therapy, mRNA is often used to try to make cells produce therapeutic proteins, and the researchers used a type of biodegradable, positively charged polymer called hyper- branched poly (beta amino esters) to try to stabilise RNA so that it can be inhaled just like asthmatic drugs are inhaled. The technique was tested by encapsulating a stretch of mRNA that encoded a fluorescent protein called luciferase and seeing if it could be expressed in the lungs of mice. The researchers synthesised 150nm diameter spherical particles of the delivery esters and experimental mRNA, and suspended them in liquid droplets which they administered to the mice in mist form through a nebuliser. Luciferase was produced inside the mice’s lungs within 24 hours and diminished as time went by, but repeated doses kept the level steady. They also succeeded in freeze drying the nanoparticles into a powder that could be administered through an inhaler as opposed to the nebuliser used in their experiment. An early-stage clinical trial of inhalable mRNA for cystic fibrosis has begun in the US.