Researchers use gene-editing technology to treat fatal lung disease before birth

Gene-editing technology has been known to treat many diseases effectively, but this time scientists have taken it a step ahead by treating a lethal disease even before birth.

A team from Children’s Hospital of Philadelphia (CHOP) and Penn Medicine has used CRIPSR gene-editing tech to kill a fatal lung disease, in which the disease causes death within hours of birth, in a mice model while still in womb.

“We wanted to know if this could work at all,” said study co-leader Edward Morrisey. “The trick was how to direct the gene-editing machinery to target cells that line the airways of the lungs.”

Scientists put human gene into monkeys to make them smarter, human-like

The team injected mice with the fatal lung disease with the gene editors late in gestation and found distinct changes in the quality of their lungs. The team demonstrated that accurately timed in-utero delivery of CRISPR gene-editing reagents to the amniotic fluid during fetal development led to targeted changes in the lungs of mice.

They introduced the gene editors into developing mice four days before birth – similar to third trimester in humans, reported Penn Medicine News. “The developing fetus has many innate properties that make it an attractive recipient for therapeutic gene editing,” said study co-leader William Peranteau.

“The ability to cure or mitigate a disease via gene editing in mid- to late gestation before birth and the onset of irreversible pathology is very exciting. This is particularly true for diseases that affect the lungs, whose function becomes dramatically more important at the time of birth,” he continued.

The use of CRISPR resulted in a survival rate of 22% for mice born with a mutation causing lung disease that previously killed all mice, as per the study published in Science Translational Medicine. For future, more studies will be directed towards increasing the efficiency of gene-editing in various parts of lungs, and also look at various mechanisms to deliver gene-editing tech to lungs.