Phase I/II Study of Intrapleural Administration of a Serotype rh.10 Replication-Deficient Adeno-Associated Virus Gene Transfer Vector Expressing the Human α1-Antitrypsin cDNA to Individuals with α1-Antitrypsin Deficiency

Gene-Based Therapy for Alpha-1 Antitrypsin Deficiency

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

Intrapleural Administration of an AAVrh.10 Vector Coding for Human α1-Antitrypsin for the Treatment of α1-Antitrypsin Deficiency

(2013) Maria J. Chiuchiolo et al.   Human Gene Therapy Clinical Development

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

Gene therapy for alpha-1 antitrypsin deficiency

(2011) T. R. Flotte et al.   HUMAN MOLECULAR GENETICS

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

Alpha1-Antitrypsin Deficiency

(2009) Edwin K. Silverman et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

Intravenous Human Plasma-Derived Augmentation Therapy in α1-Antitrypsin Deficiency: From Pharmacokinetic Analysis to Individualizing Therapy

(2008) Nuria Padullés Zamora et al.   ANNALS OF PHARMACOTHERAPY

Alpha-1 antitrypsin deficiency: A conformational disease associated with lung and liver manifestations

(2008) C. M. Greene et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Transduction Efficiencies of Novel AAV Vectors in Mouse Airway Epithelium In Vivo and Human Ciliated Airway Epithelium In Vitro

(2008) Maria P Limberis et al.   MOLECULAR THERAPY