Editing Faulty Genes Before Birth? Biomedical Tool May Help, Study Reveals
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A new study shows that a biomedical tool can successfully deliver genetic material to edit faulty genes in developing fetal brain cells. A complex transportation system with a revolutionary delivery method. The scientists found a way to deliver messenger RNA (mRNA) to cells that will be translated to functional proteins. This delivery method uses a unique lipid nanoparticle (LNP) formulation to carry mRNA. The objective is to introduce or transfect mRNA genetic material into the cells. The mRNA then would translate instructions to build proteins.
In a recent Nature Nanotechnology paper, Wang, Murthy and their team described a new lipid nanoparticle formulation to safely and efficiently deliver mRNA. lipid nanoparticle 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 lipid nanoparticle carrier, which allows the mRNA cargo to be released. The study showed that the lipid nanoparticle method is more efficient at mRNA translation, reducing the need for potentially toxic doses.
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