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Scientists have used CRISPR technology to edit out a lung-disease–causing gene while mice were in utero, rescuing the function of the lung and not affecting other organs, they reported on Wednesday (April 17) in Science Translational Medicine. Following the inactivation of the target gene, the mice survived after being born, unlike untreated animals.
Because mice carrying the disease will die soon after birth, the gene-editing intervention needed to occur before delivery, say the authors. They injected CRISPR-Cas9 into the amniotic fluid of pregnant mice whose fetuses carried a mutated version of the SFTPC gene that codes for a protein that helps prevent the lung from collapsing when emptied, states the paper.
The authors report that gene editing was successful in the target cells in the lungs of 20 percent of the mice.
“What’s exciting about this paper is that they showed specific targeting to just the affected ...
