Down syndrome could potentially be treated in the womb

According to a new Rutgers-led study, targeting a key gene in the womb could potentially lead to a treatment for Down syndrome, by reversing abnormal embryonic brain development and improving cognitive function after birth.

Typically, a baby is born with 46 chromosomes; however, babies with Down syndrome have an extra copy of chromosome 21, also known as gene OLIG2.

Subsequently, this changes how the baby’s body and brain develop and therefore can lead to mental and physical challenges, as well as, variability in cognitive development.

Currently, around one in every 1,000 babies born will have Down syndrome, in the UK alone there are 60,000 people with the condition.

However, researchers have found a way to alter the extra chromosome in a baby’s cells which cause the condition.

After extracting skill cells from Down syndrome patients and genetically reprogrammed those cells to human-induced pluripotent stem cells (hiPSCs), researchers were able to develop the 3-D brain organoid model.

As a result, researchers found that using drugs to inhibit OLIG2 stopped the production of harmful neurons.

According to the National Institutes of Health, resembling embryonic stem cells, the special cells can develop into many different types of cells, including brain cells, during early life and growth and are useful tools for drug development and disease modelling.

Peng Jiang, Assistant Professor in the Department of Cell Biology and Neuroscience at Rutgers University-New Brunswick, said: “Our results suggest the OLIG2 gene is potentially an excellent prenatal therapeutic target to reverse abnormal embryonic brain development, as well as improving postnatal cognitive function.”

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