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Unique mRNA delivery method could fix faulty genes before birth

Date: 23.10.2024 

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

Kredit: UC Davis.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.

 


 

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