Ontology highlight
ABSTRACT:
SUBMITTER: Carlon MS
PROVIDER: S-EPMC4064737 | biostudies-literature | 2014 Jun
REPOSITORIES: biostudies-literature
Carlon Marianne S MS Vidović Dragana D Dooley James J da Cunha Marina Mori MM Maris Michael M Lampi Youlia Y Toelen Jaan J Van den Haute Chris C Baekelandt Veerle V Deprest Jan J Verbeken Erik E Liston Adrian A Gijsbers Rik R Debyser Zeger Z
Human gene therapy 20140326 6
Gene therapy of the lung has the potential to treat life-threatening diseases such as cystic fibrosis and α(1)-antitrypsin or surfactant deficiencies. A major hurdle for successful gene therapy is the development of an immune response against the transgene and/or viral vector. We hypothesized that by targeting the airways in the perinatal period, induction of an immune response against the vector particle could be prevented because of immaturity of the immune system, in turn allowing repeated ge ...[more]