Breakthrough in Gene Editing Offers Hope for In-Utero Treatment of Neurodevelopmental Disorders
Researchers have developed a novel method using lipid nanoparticles to deliver gene-editing mRNA to fetal brain cells, potentially preventing conditions like Angelman syndrome before birth.
- The new technology successfully delivers Cas9 mRNA to fetal brain cells using lipid nanoparticles, allowing for gene editing in developing neurons.
- In tests on mice, the method achieved gene edits in 30% of brain stem cells, with significant transfection in the hippocampus and cortex.
- Early intervention could prevent the progression of neurodevelopmental disorders, offering a potential cure before symptoms appear post-birth.
- The study, a collaboration between UC Davis and UC Berkeley, highlights the efficiency and reduced toxicity of this delivery method.
- Researchers aim to develop this approach for treating genetic disorders detectable through prenatal testing, revolutionizing prenatal care.
ScienceDaily 
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