Wednesday, March 27, 2019

Gene therapy for unborn babies

The Guardian posted an article about doctor's using CRISPR to treat a rare brain disorder in unborn babies. The gene therapy they are using could mend the mutations in the womb that causes Angelman Syndrome. The treatment has never been tested before but it would involve the doctor's injecting a virus into the brain that infects the neurons and delivers molecules to correct the genetic faults. The disorder can be detected at 10 weeks and has been tested on a brain cell grown in a dish.  It is suggested that the procedure will be best performed in the second trimester. Normal brain development gene comes from UBE3A where the gene from the mother is "on" and from the father is silenced. During development the gene from the mother is missing or mutated, so the CRISPR technology turns the father's gene "on".
Angelman syndrome affects one in 15,000 births and the children often experience seizures, along with difficulty sleeping and walking. I believe that once this is tested more that it would greatly improve the lives of many children. The testing could be better if doctor's were able to actually use the CRISPR technology on babies in the womb but due to the uncertainty I understand why the testing is only done on mice. Hopefully within the next few years this technology and syndrome can be handled and cured or at least be a treatment for children already suffering from the syndrome. 

1 comment:

  1. This article just further shows the capabilities of CRISPR technology and genetic testing. Just a few years ago, it would not have been known during pregnancy that the baby had a medical condition like Angelman Syndrome. With the advancements in technology, the diagnosis and potential treatment option can be discussed. It is very interesting that the syndrome can be corrected by injecting a virus into the developing brain. However, extensive research needs to be conducted before beginning trials in humans because of the potential ramifications of it not working or something going wrong.