PMID: 2117165Jan 1, 1990Paper

Strategies for aerosol therapy of alpha 1-antitrypsin deficiency by the aerosol route

Lung
R C Hubbard, R G Crystal

Abstract

Alpha 1-antitrypsin (AAT) deficiency is a genetic disease in which low serum and lung levels of the antiprotease AAT cause a deficiency of the anti-elastase defensive screen of the lower respiratory tract such that neutrophil elastase is free to degrade the connective tissue of the lung, eventually resulting in emphysema. Intravenous AAT infusion therapy restores lung levels of AAT, but is inefficient, costly and a demanding form of therapy. As an alternative, we evaluated aerosol delivery of human plasma AAT (pAAT) and recombinant DNA-produced AAT (rAAT), as a means of providing anti-elastase protection to the lower respiratory tract. In vitro studies demonstrated that both pAAT and rAAT can be aerosolized into droplets suitable for alveolar deposition without loss of antiprotease activity. When administered by aerosol to individuals with AAT deficiency, pAAT and rAAT each significantly raised lung epithelial lining fluid levels of AAT and anti-neutrophil elastase capacity, with the likelihood that twice daily administration of 100 mg of either form would result in normalization of lung anti-elastase defenses at the alveolar surface. Studies in sheep further demonstrated that the aerosolized pAAT and rAAT were each able to pas...Continue Reading

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Citations

Jul 29, 2020·Frontiers in Pharmacology·Srinu TumparaSabina Janciauskiene
Jan 3, 1996·Journal of Aerosol Medicine : the Official Journal of the International Society for Aerosols in Medicine·A R Brown, J A Pickrell
Aug 24, 2005·The Journal of Immunology : Official Journal of the American Association of Immunologists·Brice KorkmazFrancis Gauthier
Jun 6, 2013·Expert Review of Respiratory Medicine·Judith A Brebner, Robert A Stockley
Nov 5, 2014·Expert Opinion on Biological Therapy·Heather S Loring, Terence R Flotte
Nov 5, 2016·Expert Review of Respiratory Medicine·Maria Paula Henao, Timothy J Craig
Sep 26, 2019·Expert Opinion on Investigational Drugs·Anita Pye, Alice M Turner
Mar 24, 2020·Human Gene Therapy·Ronald G Crystal
Aug 21, 1992·Pharmaceutisch Weekblad. Scientific Edition·A Van der Kuy
Jun 15, 2006·Amino Acids·E KarnaukhovaB Golding
Aug 3, 2007·Respiration; International Review of Thoracic Diseases·Alice M Wood, Robert A Stockley
Dec 25, 2010·Nanomedicine·Fernanda AndradeBruno Sarmento
May 1, 1998·American Journal of Respiratory and Critical Care Medicine·R J Mason, R G Crystal
Nov 11, 1999·American Journal of Respiratory and Critical Care Medicine·P J Barnes
Jan 15, 2008·Journal of Inherited Metabolic Disease·C M GreeneN G McElvaney
Feb 4, 2006·Current Opinion in Pulmonary Medicine·Hatem Abusriwil, Robert A Stockley
Apr 14, 2010·Journal of Aerosol Medicine and Pulmonary Drug Delivery·David E Geller, Kenneth C Kesser
Jun 19, 2013·BioDrugs : Clinical Immunotherapeutics, Biopharmaceuticals and Gene Therapy·Alice M Turner
Aug 8, 2013·Therapeutic Delivery·Mariam Ibrahim, Lucila Garcia-Contreras
Jan 20, 2015·Expert Review of Respiratory Medicine·Alessandro N FranciosiNoel Gerry McElvaney
Dec 9, 1993·Journal of Aerosol Medicine : the Official Journal of the International Society for Aerosols in Medicine·R E Wood, M R Knowles
May 9, 2009·FASEB Journal : Official Publication of the Federation of American Societies for Experimental Biology·Sadaf SohrabIrina Petrache

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