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"The developing fetus has many innate properties that make it an attractive recipient for therapeutic gene editing," said study co-leader William H. Peranteau, MD, an investigator at CHOP's Center for Fetal Research, and a pediatric and fetal surgeon in CHOP's Center for Fetal Diagnosis and Treatment. "Furthermore, 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."
The lung conditions the team is hoping to solve -- congenital diseases such as surfactant protein deficiency, cystic fibrosis, and alpha-1 antitrypsin -- are characterized by respiratory failure at birth or chronic lung disease with few options for therapies. About 22 percent of all pediatric hospital admissions are because of respiratory disorders, and congenital causes of respiratory diseases are often lethal, despite advances in care and a deeper understanding of their molecular causes. Because the lung is a barrier organ in direct contact with the outside environment, targeted delivery to correct defective genes is an attractive therapy.
Study - Co-leader - Edward - E - Morrisey
"We wanted to know if this could work at all," said study co-leader Edward E. Morrisey, PhD, a professor of Cardiovascular Medicine in the Perelman School of Medicine at the University of Pennsylvania. "The trick was how to direct the gene-editing machinery to target cells that line the airways of the lungs."
The researchers showed that precisely timed in utero delivery of CRISPR gene-editing reagents to the amniotic fluid during fetal development resulted in targeted changes in the lungs of mice. They introduced the gene editors into developing mice four days before birth, which is analogous to the third trimester in humans.
Cells - Percentage
The cells that showed the highest percentage of editing were...
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