Using CRISPR gene editing technology, a team from Children’s Hospital of Philadelphia and Penn Medicine have managed to edit a harmful mutation that causes death within hours after birth, in an early proof of concept study with mice.
The gene in question is responsible for creating a surfactant in the lungs, which is part of a protective film that shields the lungs’ alveoli from being collapsed due to the fluid mechanics of water particles, as well as the intrusion of bacteria and viruses. Genetic surfactant deficiency is a mutation that causes nearly instant death due to the lack of this protective film.
The researchers showed that by applying the CRISPR gene-editing technique to the amniotic fluid during the equivalent of the third trimester, 22 percent of the mice that had the genetic mutation managed to survive and develop normal looking lungs. Whereas, without the treatment, all of the mice would have died within hours.
Why 22%? well, CRISPR technology is still under development, and can make mistakes in which genes it edits at this time. However, some groups are working on improving the accuracy, and we believe a host of genetic diseases will be curable using the technology in the future.