Gene Therapy May Help Treat Rare Brain Disorder in Unborn Babies

NORTH CAROLINA – Scientists are going to conduct a gene therapy which could help to treat mental disorders in unborn babies. This process will involve the mending mutations in the brains of foetuses in the womb. It is a new attempt by scientists and it would involve the injecting of a harmless virus to infect the neurons and delivering a set of molecules to correct genetic faults in unborn babies. The right time of performing this therapy will be during the initial stage of child development. The second trimester is the stage when it is possible to remove genetic disorders using this therapy.

The mental disorders, known as Angelman syndrome is rare to be found in childbirths. Children with this genetic disorder have difficulty with walking and sleeping. Also, they could spend the whole of their lives without uttering a word. The sleeping problems due to this genetic disorder are such that parents lock their affected children in a room in order to prevent their accidents due to their walking during sleeping.

In a normal person, there are always two copies of genes present in their body, each inherited from mother and father. For normal brain development, the mother’s gene, UBE3A has to be switched on while there should no switching of the father’s gene. In Angelman Syndrome, either mother copy of the gene, UBE3A is missing or mutated. And with the use of gene therapy, father’s gene is switched on using a version of a powerful gene editing tool known as CRISPR-Cas9.

Mark Zylka, a neurologist at the University of North Carolina while speaking at an American Association for the Advancement of Science meeting in Washington DC said the experiment has been performed on mice. During the studies, it was found that when the gene therapy is given in the womb, it activates UBE3A in key parts of the brain. This therapy switched the gene on in the cortex, hippocampus, and the cerebellum part of the brain which eventually helps in ensuring specific functions of the different parts of the brain.

Zylka pointed out that although no one has tried this gene therapy in the fetus in the womb, still there is a huge possibility for this to work well. He said if this happens then it would be easy to cure neurodevelopment disorders in the future.