UMMS Affiliation
Gene Therapy Center; Department of Pediatrics
Publication Date
6-2012
Document Type
Article
Subjects
Gene Therapy; Stem Cell Transplantation; Genetic Diseases, Inborn; Lung Diseases; Cystic Fibrosis; alpha 1-Antitrypsin Deficiency; Sudden Infant Death
Disciplines
Congenital, Hereditary, and Neonatal Diseases and Abnormalities | Genetics and Genomics | Pediatrics | Respiratory Tract Diseases
Abstract
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.
Rights and Permissions
Copyright © 2012 Mary Ann Liebert, Inc.; Human Gene Therapy is available online at: http://online.liebertpub.com. Publisher PDF posted as allowed by the publisher's author rights policy at http://www.liebertpub.com/archpolicy/hgtb.
DOI of Published Version
10.1089/hum.2012.087
Source
Hum Gene Ther. 2012 Jun;23(6):548-56. Link to article on publisher's site
Journal/Book/Conference Title
Human gene therapy
Related Resources
PubMed ID
22642257
Repository Citation
Keeler, Allison M. and Flotte, Terence R., "Cell and gene therapy for genetic diseases: inherited disorders affecting the lung and those mimicking sudden infant death syndrome" (2012). Pediatric Publications and Presentations. 15.
https://escholarship.umassmed.edu/peds_pp/15
Included in
Congenital, Hereditary, and Neonatal Diseases and Abnormalities Commons, Genetics and Genomics Commons, Pediatrics Commons, Respiratory Tract Diseases Commons