New mRNA Delivery Technique Could Correct Genetic Disorders Before Birth

In a new study from UC Davis and UC Berkeley, researchers have developed a method to deliver mRNA to fetal brain cells, potentially offering a way to correct genetic disorders like Angelman and Rett syndrome before birth. By injecting messenger RNA (mRNA) encapsulated in lipid nanoparticles (LNPs) directly into fetal brain cells, the study showed promising results in a mouse model for gene editing in the developing brain, reported in ACS Nano.

Understanding the Technology

The LNP technology used here acts as a “delivery vehicle” for mRNA, which serves as a blueprint for proteins in cells. By delivering mRNA that instructs the cell to build the Cas9 enzyme, which can edit genes, researchers successfully targeted cells in the fetal brain. This method could correct genetic mutations at a critical developmental stage, potentially stopping harmful cellular changes before birth. Senior researcher Dr. Aijun Wang, a UC Davis biomedical engineering professor, noted that this approach could allow for the correction of faulty genes during a crucial window in fetal brain development.

Implications and Benefits of In Utero Gene Editing

The research provides a model for how neurodevelopmental conditions could be treated early, before the blood-brain barrier forms, which usually limits treatments after birth. Correcting cells early could lead to more lasting benefits, particularly since a significant number of neurons in areas critical for memory and cognition were shown to incorporate the healthy genetic material.

Future of LNP mRNA Treatments

The LNP method also minimises the risks of inflammation, a common challenge in mRNA delivery to the brain. This research lays the groundwork for future therapies targeting the central nervous system, where treatments given in utero could translate into better health outcomes at birth. The researchers plan to continue developing this technology to see if it can be applied more widely in disease prevention before birth, with potential broader applications for other genetic conditions.