Ontology highlight
ABSTRACT:
SUBMITTER: Alapati D
PROVIDER: S-EPMC6822403 | biostudies-literature | 2019 Apr
REPOSITORIES: biostudies-literature
Alapati Deepthi D Zacharias William J WJ Hartman Heather A HA Rossidis Avery C AC Stratigis John D JD Ahn Nicholas J NJ Coons Barbara B Zhou Su S Li Hiaying H Singh Kshitiz K Katzen Jeremy J Tomer Yaniv Y Chadwick Alexandra C AC Musunuru Kiran K Beers Michael F MF Morrisey Edward E EE Peranteau William H WH
Science translational medicine 20190401 488
Monogenic lung diseases that are caused by mutations in surfactant genes of the pulmonary epithelium are marked by perinatal lethal respiratory failure or chronic diffuse parenchymal lung disease with few therapeutic options. Using a CRISPR fluorescent reporter system, we demonstrate that precisely timed in utero intra-amniotic delivery of CRISPR-Cas9 gene editing reagents during fetal development results in targeted and specific gene editing in fetal lungs. Pulmonary epithelial cells are predom ...[more]