firstwordpharmaApril 21, 2019
Tag: CRISPR , lung diseases , Birth
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.
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.
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.
They introduced the gene editors into developing mice four days before birth, which is analogous to the third trimester in humans.
The cells that showed the highest percentage of editing were alveolar epithelial cells and airway secretory cells lining lung airways, the news source said.
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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