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CRISPR edits lung-disease gene in utero, hitting only the affected organ in a mouse study

7 days ago   |   By STAT

Companies that hope to treat severe inherited diseases via CRISPR genome editing are already testing the technique in adults, while push-the-envelope types are arguing for repairing defective genes much earlier - in IVF embryos so new they're still in a lab dish. Now scientists in Philadelphia have taken preliminary steps toward a possible middle way: They injected CRISPR into the amniotic fluid of pregnant mice, editing a lung-disease-causing gene in a small number of mouse fetuses, they reported on Wednesday.
In utero editing offers advantages for at least some diseases, said Dr. William...
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