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Researchers writing in Science Translational Medicine say they used CRISPR gene editing to thwart a lethal lung disease in an animal model in which a harmful mutation causes death within hours after birth, reported ScienceDaily.
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According to the news source, the proof-of-concept study showed that in utero editing could be a promising new approach for treating lung diseases before birth.
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Specifically, investigators 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.
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They introduced the gene editors into developing mice four days before birth, which is analogous to the third trimester in humans.
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The cells that showed the highest percentage of editing were alveolar epithelial cells and airway secretory cells lining lung airways, the news source said.
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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.
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