Scientists are developing a new gene therapy that could cure a devastating medical brain disorders in unborn babies by using Crispr-Cas9 tool.
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.
“We believe that this could provide a treatment, if not a cure, depending on when it’s injected,” said Mark Zylka, a neurobiologist at the University of North Carolina.
The therapy is aimed at a rare brain disorder known as Angelman syndrome, which affects one in 15,000 births. Zylka said children with Angelman syndrome can have such severe sleeping difficulties.
Healthy people tend to have two copies of every gene in the genetic code, one inherited from their mother and the other from their father. But both copies are not always switched on. For normal brain development, the mother’s copy of a gene called UBE3A is switched on, while the father’s copy is silenced.
In Angelman syndrome, the mother’s copy of UBE3A is either missing or mutated, meaning there is no UBE3A active at all. Zylka’s therapy uses a version of a powerful gene editing tool known as Crispr-Cas9 to switch the father’s copy back on.