Ontology highlight
ABSTRACT:
SUBMITTER: Janosz E
PROVIDER: S-EPMC8455329 | biostudies-literature | 2021 Sep
REPOSITORIES: biostudies-literature
Janosz Ewa E Hetzel Miriam M Spielmann Hanna H Tumpara Srinu S Rossdam Charlotte C Schwabbauer Marc M Kloos Doreen D von Kaisenberg Constantin C Schambach Axel A Buettner Falk F R FFR Janciauskiene Sabina S Lachmann Nico N Moritz Thomas T
Gene therapy 20210719 9
Inherited deficiency of the antiprotease alpha-1 antitrypsin (AAT) is associated with liver failure and early-onset emphysema. In mice, in vivo lentiviral transduction of alveolar macrophages (AMs) has been described to yield protective pulmonary AAT levels and ameliorate emphysema development. We here investigated the pulmonary transplantation of macrophages (PMT) transgenic for AAT as a potential therapy for AAT deficiency-associated lung pathology. Employing third-generation SIN-lentiviral ve ...[more]