Ontology highlight
ABSTRACT:
SUBMITTER: Stiles KM
PROVIDER: S-EPMC6361473 | biostudies-literature | 2018 Aug
REPOSITORIES: biostudies-literature
Stiles Katie M KM Sondhi Dolan D Kaminsky Stephen M SM De Bishnu P BP Rosenberg Jonathan B JB Crystal Ronald G RG
Chronic obstructive pulmonary diseases (Miami, Fla.) 20180817 4
Alpha-1 antitrypsin deficiency (AATD) manifests primarily as early-onset emphysema caused by the destruction of the lung by neutrophil elastase due to low amounts of the serine protease inhibitor alpha-1 antitrypsin (AAT). The current therapy involves weekly intravenous infusions of AAT-derived from pooled human plasma that is efficacious, yet costly. Gene therapy applications designed to provide constant levels of the AAT protein are currently under development. The challenge is for gene therap ...[more]