Unique mRNA delivery method could fix faulty genes before birth

A new study shows that a biomedical tool can successfully deliver genetic material to edit faulty genes in developing fetal brain cells.

The technology, tested in mice, might have the potential to stop the progression of genetic-based neurodevelopmental conditions, such as Angelman syndrome and Rett syndrome, before birth.

"The implications of this tool for treating neurodevelopmental conditions are profound. We can potentially correct genetic anomalies at a foundational level during critical periods of brain development," said the study's senior author Aijun Wang, a UC Davis professor of surgery and biomedical engineering.

Instead of delivering proteins, scientists found a way to deliver mRNA to cells that will be translated to functional proteins within the cells. This delivery method uses a unique lipid nanoparticle (LNP) formulation to carry mRNA. The objective is to introduce (transfect) mRNA genetic material into the cells. The mRNA would then translate instructions to build proteins.

In a recent Nature Nanotechnology paper, Wang, Murthy and their team described a new LNP formulation to safely and efficiently deliver mRNA. LNPs carrying mRNA need to arrive at the cells, where they will be taken in through a process known as endocytosis. There, the cell breaks the LNP carrier, which allows the mRNA cargo to be released.

Image source: UC Davis.