In utero lung gene editing sftpc

The objectives of this proof-of-concept study were to evaluate, in mouse models, the feasibility and efficiency of prenatal lung gene editing after intra-amniotic delivery of CRISPR-Cas9 via adenoviral vector and to demonstrate that prenatal pulmonary gene editing can alter the phenotype of a perinatal lethal monogenic lung disease. These studies demonstrating the feasibility of prenatal gene editing with high specificity for the lung represent a novel and promising approach to address the unmet need for new therapeutic approaches to congenital lung diseases that are fatal at birth.