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Systemic In Utero Gene Editing As a Treatment for Cystic Fibrosis
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Systemic In Utero Gene Editing As a Treatment for Cystic Fibrosis
Researchers demonstrated cystic fibrosis (CF) transmembrane conductance regulator gene correction in multiple tissues affected by CF after a single in utero administration of nanoparticles containing gene editing cargo. [Proceedings of The National Academy of Sciences of The United States of America] Abstract
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