Authors:

Jessica Wong

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About Paper:

In utero mRNA-based treatments offer promising care for treating central nervous system (CNS) disorders before disease onset but there is a lack of safe and effective delivery vehicles. We demonstrate that densely PEGylated lipid nanoparticles (ADP- LNP) can serve as a platform for development of in utero mRNA- based therapeutics. ADP-LNPs containing Cre-mRNA were administered to fetal Ai9 mice through in utero intracerebroventricular (ICV) injection and a cesarean section was performed 48 hours later. Flow cytometry showed that 29.3% of the fetal mouse brain cells were td-tomato positive and therefore had been successfully transfected. Additionally, immunohistochemistry (IHC) staining was performed and showed that the majority of edited cells are Sox2+, Nestin+, and Ki67+ neural stem cells. These results confirm the specificity and efficacy of ADP-LNP-mediated mRNA delivery to target progenitor cell populations within the fetal brain. These target cells have the potential to proliferate and expand to generate high levels of transfection as the brain develops, offering this as a promising treatment for neurodevelopmental disorders. Defining the Estrogen Synthesis Pathway in the Zebrafish Ovary Melinda Wong

Source:

UC Davis / Biomedical Engineering / 2024

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No topics listed

Co-authors:

Jessica Wong