Gene Therapy Could Treat Rare Brain Disorder in Unborn Babies

February 20, 2019

(The Guardian) – Scientists are developing a radical form of gene therapy that could cure a devastating medical disorder by mending mutations in the brains of foetuses in the womb. The treatment, which has never been attempted before, would involve doctors injecting the feotus’s brain with a harmless virus that infects the neurons and delivers a suite of molecules that correct the genetic faults. Tests suggest that the therapy will be most effective around the second trimester, when their brains are in the early stages of development.

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