Gene Editing Offers Treatment For Deadly Disease That Turns Babies Blue

The gene editing tool CRISPR/Cas9 continues to provide scientists new ways to understand and fight previously untreatable diseases. A new study offers a potential treatment for a deadly respiratory disorder that affects babies.



Most cases of Alveolar Capillary Dysplasia with Misalignment of Pulmonary Veins (ACDMPV) are untreatable. The most common sign of the disease is when the lips and skin of babies turn blue.

ACDMPV cuts flow of oxygen in the pulmonary system as the lungs blood vessels on the lungs fail to properly develop during pregnancy. The disease can lead to hypoxia, inflammation and death.

To date, the only effective way to treat the disease is lung transplant, according to Vlad Kalinichenko, lead researcher from the Cincinnati Children's Perinatal Institute Center for Lung Regenerative Medicine.

American Journal of Respiratory and Critical Care Medicine, suggests that there may be a less invasive way to fight ACDMPV.

"There are no effective treatments other than a lung transplant, so the need for new therapeutics is urgent," Kalinichenko said in a statement. "We identified a nanoparticle therapeutic strategy to increase the number of alveolar capillaries and help preserve respiratory function for at least a subset of the babies with this congenital lung disease."

For the study, the researchers used mouse subjects to mimic the disease. The team was able to identify how it develops, which helped them create a potential treatment.

The researchers proposed that a nanoparticle technology can deliver a protein called STAT3 to the lungs of babies, which could then trigger the development of blood vessels in the lungs. The treatment uses a gelatin-like material that can travel to different parts of the body.

Lab tests showed that providing STAT3 to newborn mice effectively restored the development of pulmonary blood vessels.

Kalinichenko said a multicenter clinical trial is required to confirm the effects of their treatment for ACDMPV.

If the nanoparticles is confirmed effective in future clinical trials, the treatment could be considered for STAT3 gene therapy in infants with ACDMPV, the researcher added.






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