Every day, scientists and medical researchers finetune previous technology and techniques, or they find new applications for existing ones. Recently, a team from Tufts University, with collaboration from Harvard Medical School and the University of Massachusetts, Boston, made a new application for lipid-based nanoparticles (LNPs) that could potentially treat a multitude of diseases.
What are LNPs?
LNPs are essentially a delivery system, in this case designed to latch on to specific types of tissue in the body. They saw their first use in the development of two major mRNA vaccines, which had enormous success. With how specifically doctors can program LNPs, they can be used to deliver gene treatment to almost any part of the body and feasibly treat numerous conditions.
Treating a rare lung condition
The Tufts team used these LNPs to treat a rare lung condition called lymphangioleiomyomatosis which basically causes smooth lung tissue to grow uncontrollably and mutate, creating harmful cysts in the lungs. The gene involved that gets mutated is Tsc2. In a mouse case, the team packed LNPs with mRNA, which was designed to provide new instructions for the Tsc2 gene and stop it from harmfully mutating.
The results were very promising. The team saw a significant reduction in the development of cysts in the lungs in the mouse case they performed.
“The effect was quite impressive,” says Qiaobing Xu, professor of biomedical engineering at Tufts. “The mRNA-packed LNPs could be a promising intervention for this awful disease, and we hope to carry it further into clinical trials.”
Basically, this new method is like providing code to genes, telling them how to behave. Think of it as providing temporary instructions to genes. It is temporary and fades with time, requiring more doses, but the research is promising. This could potentially be a way into treating many harmful diseases.