In-Utero Gene Editing Could Halt Neurodevelopmental Disorders

A new study has developed a breakthrough biomedical tool that can deliver genetic material to edit faulty genes in fetal brain cells. Tested in mice, this technology offers the potential to stop the progression of neurodevelopmental conditions, like Angelman and Rett syndromes, before birth.

The researchers used lipid nanoparticles (LNPs) to deliver Cas9 mRNA, enabling gene editing within developing neurons. This innovative approach may one day help treat genetic disorders in-utero, preventing severe symptoms from developing after birth.

Key Facts:

  • LNPs successfully deliver Cas9 mRNA to fetal brain cells, enabling gene edits.
  • The tool was able to transfect 30% of brain stem cells, allowing wide impact.
  • Early intervention could prevent neurodevelopmental disorders like Angelman syndrome.

To learn more, read the full article: https://neurosciencenews.com/eurodevelopment-gene-editing-27938/?fbclid=IwY2xjawGfFGtleHRuA2FlbQIxMQABHSVYl70pPTGhPD6w1MJsaZHjkK2dfDjpcQ6q3o0gyhlxb4KGdtevR9U28A_aem_UEtFuXdp3EblLEkaQImtOw

Author: Nadine Yehya
Source: UC Davis