Research ArticleGENE EDITING

In utero gene editing for monogenic lung disease

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Science Translational Medicine  17 Apr 2019:
Vol. 11, Issue 488, eaav8375
DOI: 10.1126/scitranslmed.aav8375

Editing out a lethal lung disease

Surfactant, a lipoprotein mixture that reduces lung surface tension, is essential for normal lung function. In rare cases, infants are born with genetic surfactant deficiency, resulting in rapid death from respiratory failure. Because of the immediate perinatal fatality associated with this disease, any effective intervention would need to be applied before delivery. Alapati et al. used a mouse model of genetic surfactant deficiency to demonstrate the feasibility of in utero gene editing to delete the mutant allele. The authors showed that correction of the genetic defect before birth improved lung development and survival in the treated animals, demonstrating the feasibility of this therapeutic intervention.

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