Gene Therapy Center; Department of Pediatrics
Medical Subject Headings
Gene Therapy; Stem Cell Transplantation; Genetic Diseases, Inborn; Lung Diseases; Cystic Fibrosis; alpha 1-Antitrypsin Deficiency; Sudden Infant Death
Congenital, Hereditary, and Neonatal Diseases and Abnormalities | Genetics and Genomics | Pediatrics | Respiratory Tract Diseases
Some of the first human gene therapy trials targeted diseases of the lung and provided important information that will continue to help shape future trials. Here we describe both cell and gene therapies for lung diseases such as cystic fibrosis and alpha-1 antitrypsin disorder as well as fatty acid oxidation disorders that mimic sudden infant death syndrome (SIDS). Human clinical gene therapy trials for cystic fibrosis and alpha-1 antitrypsin have been performed using a variety of vectors including adenovirus, adeno-associated virus, and nonviral vectors. No human clinical gene therapy trials have been performed for disorders of fatty acid oxidation; however, important proof-of-principle studies have been completed for multiple fatty acid oxidation disorders. Important achievements have been made and have yet to come for cell and gene therapies for disorders of the lung and those mimicking SIDS.