Current status of gene therapy for α-1 antitrypsin deficiency

The Circulating Proteinase Inhibitor  -1 Antitrypsin Regulates Neutrophil Degranulation and Autoimmunity

(2014) D. A. Bergin et al.   Science Translational Medicine

Gene-Based Therapy for Alpha-1 Antitrypsin Deficiency

(2013) Christian Mueller et al.   COPD-Journal of Chronic Obstructive Pulmonary Disease

The Electrophoretic α1-Globulin Pattern of Serum in α1-Antitrypsin Deficiency

(2013) Carl-Bertil Laurell et al.   COPD-Journal of Chronic Obstructive Pulmonary Disease

Airway Disease in Alpha-1 Antitrypsin Deficiency

(2013) Charlie Strange   COPD-Journal of Chronic Obstructive Pulmonary Disease

Human Treg responses allow sustained recombinant adeno-associated virus–mediated transgene expression

(2013) Christian Mueller et al.   JOURNAL OF CLINICAL INVESTIGATION

Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial

(2012) D Gaudet et al.   GENE THERAPY

Sustained miRNA-mediated Knockdown of Mutant AAT With Simultaneous Augmentation of Wild-type AAT Has Minimal Effect on Global Liver miRNA Profiles

(2012) Christian Mueller et al.   MOLECULAR THERAPY

A Review of α1-Antitrypsin Deficiency

(2011) James K. Stoller et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α1-Antitrypsin: Interim Results

(2011) Terence R. Flotte et al.   HUMAN GENE THERAPY

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

(2011) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Preclinical Evaluation of a Recombinant Adeno-Associated Virus Vector Expressing Human Alpha-1 Antitrypsin Made Using a Recombinant Herpes Simplex Virus Production Method

(2010) Jeffrey D. Chulay et al.   HUMAN GENE THERAPY

Amelioration of emphysema in mice through lentiviral transduction of long-lived pulmonary alveolar macrophages

(2009) Andrew A. Wilson et al.   JOURNAL OF CLINICAL INVESTIGATION

Dual Reporter Comparative Indexing of rAAV Pseudotyped Vectors in Chimpanzee Airway

(2009) Terence R Flotte et al.   MOLECULAR THERAPY

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

(2009) M. L. Brantly et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Clinical gene therapy using recombinant adeno-associated virus vectors

(2008) C Mueller et al.   GENE THERAPY

Apparently Nonspecific Enzyme Elevations After Portal Vein Delivery of Recombinant Adeno-Associated Virus Serotype 2 Vector in Hepatitis C Virus-Infected Chimpanzees

(2008) Terence R. Flotte et al.   HUMAN GENE THERAPY

Recombinant AAV Serotype and Capsid Mutant Comparison for Pulmonary Gene Transfer of α-1-Antitrypsin Using Invasive and Noninvasive Delivery

(2008) Rejean Liqun Wang et al.   MOLECULAR THERAPY