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 …

Although our understanding of the genetics and pathology of congenital lung diseases such
as surfactant protein deficiency, cystic fibrosis, and alpha-1 antitrypsin deficiency is …

Lung alveoli, which are unique to air-breathing organisms, have been challenging to
generate from pluripotent stem cells (PSCs) in part because there are limited model systems …

Genome-editing systems based on clustered, regularly interspaced, short palindromic
repeat (CRISPR)/associated protein (CRISPR/Cas), are emerging as a revolutionary …

Pulmonary surfactant is critically important to prevent atelectasis by lowering the surface
tension of the alveolar lining liquid. While respiratory distress syndrome (RDS) is common in …

In utero gene editing has the potential to prenatally treat genetic diseases that result in
significant morbidity and mortality before or shortly after birth. We assessed the viral vector …

Genome editing in the lung has the potential to provide long-term expression of therapeutic
protein to treat lung genetic diseases. Yet efficient delivery of CRISPR to the lung remains a …

Abstract Approximately 10% of Cystic Fibrosis (CF) patients, particularly those with CF
transmembrane conductance regulator (CFTR) gene nonsense mutations, lack effective …

Since CRISPR/Cas9 was harnessed to edit DNA, the field of gene therapy has witnessed
great advances in gene editing. New avenues were created for the treatment of diseases …

Abstract Mucopolysaccharidosis type I (MPS I) is caused by deficiency of alpha-L-
iduronidase (IDUA), leading to multisystemic accumulation of glycosaminoglycans (GAG) …