A New Gene Therapy Developed To Treat A Brain Disorder In Unborn Children

Aadhya Khatri - Mar 01, 2019

This therapy can switch on a silencing gene at the early stages of the brain's development.

Scientists Used Gene Therapy To Cure Deafness In Mice

A gene therapy that can cure brain’s mutations, which lead to a severe illness in babies is currently undergoing development. This treatment is projected to take place when the child is still in the mother’s womb.

To perform this treatment, a virus is transferred into the fetus’s brain. It brings along molecules that can fix the genetic flaws.

After conducting tests, scientists conclude that the treatment will reach its full potential when done on the period between week 13 to week 28 of the mother’s pregnancy, which is when the fetus’ brain is starting to develop.

This therapy is to cure Angelman syndrome, which happens on one in 15,000 babies. Victims of this syndrome usually have smaller brains than usual, suffer from seizures, and often experience difficulty when they sleep or walk. In addition, they might not talk at all for their whole life.

Mark Zylka, University of North Carolina’s neurobiologist said that the sleeping disorder can be so serious that many parents think they have to confine the children in the room to prevent them from getting accidents.

The gene of a typical person has one from the father and another from the mother, which is called the UBE3A. This copy is essential for the child to have a normal brain. Children with Angelman syndrome have mutated UBE3A or even have no UBE3A at all. To fix this flaw, the gene therapy makes use of a variation of CRISPR-Cas9, a tool that can edit gene to activate the father’s gene.

014 2 — People with Angelman syndrome might never speak at all

When injected on mice, Zylka shared that the molecules worked on the cortex, which is responsible for actions and thoughts, the hippocampus, the part that supports memory, and the cerebellum, which regulates motor movements. The therapy also proves its effect on lab-grown human brain cells.

If this therapy works, it can open up a whole new way of treating other illnesses.

Angelman syndrome can be diagnosed when the fetus is 10 weeks old, but if it has the gene mutation, there is nothing can be done to save it. This gene therapy might be the answer to this tough issue.