CRISPR Gene Editing Successfully Treats Lethal Monogenic Lung Disease in Utero

Am J Med Genet A. 2019 Jul;179(7):1116. doi: 10.1002/ajmg.a.61213.
No abstract available

MeSH terms

  • Animals
  • CRISPR-Cas Systems
  • Disease Models, Animal
  • Epithelial Cells / metabolism
  • Epithelial Cells / pathology
  • Fetus
  • Gene Editing / methods*
  • Gene Expression
  • Genetic Therapy / methods*
  • Humans
  • Lung / metabolism*
  • Lung / pathology
  • Mice
  • Mutation
  • Proof of Concept Study
  • Pulmonary Surfactant-Associated Protein C / genetics*
  • Pulmonary Surfactant-Associated Protein C / metabolism
  • Respiratory Insufficiency / genetics*
  • Respiratory Insufficiency / metabolism
  • Respiratory Insufficiency / pathology
  • Respiratory Insufficiency / therapy*
  • Respiratory Mucosa / metabolism
  • Respiratory Mucosa / pathology

Substances

  • Pulmonary Surfactant-Associated Protein C
  • Sftpc protein, mouse