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

Engineering Liver-detargeted AAV9 Vectors for Cardiac and Musculoskeletal Gene Transfer

(2011) Nagesh Pulicherla et al.   MOLECULAR THERAPY

Adeno-Associated Virus Serotype 6 Capsid Tyrosine-to-Phenylalanine Mutations Improve Gene Transfer to Skeletal Muscle

(2010) Chunping Qiao et al.   HUMAN GENE THERAPY

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

Expression of Human α1-Antitrypsin in Mice and Dogs Following AAV6 Vector-mediated Gene Transfer to the Lungs

(2010) Christine L Halbert et al.   MOLECULAR THERAPY

Scalable Recombinant Adeno-Associated Virus Production Using Recombinant Herpes Simplex Virus Type 1 Coinfection of Suspension-Adapted Mammalian Cells

(2009) Darby L. Thomas et al.   HUMAN GENE THERAPY

Lack of Immunotoxicity After Regional Intravenous (RI) Delivery of rAAV to Nonhuman Primate Skeletal Muscle

(2009) Alice Toromanoff et al.   MOLECULAR THERAPY

Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle

(2009) Aravind Asokan et al.   NATURE BIOTECHNOLOGY

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

Intramuscular Administration of AAV1-Lipoprotein Lipase S447X Lowers Triglycerides in Lipoprotein Lipase–Deficient Patients

(2008) Erik S. Stroes et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

An efficient rHSV-based complementation system for the production of multiple rAAV vector serotypes

(2008) W Kang et al.   GENE THERAPY

Safety and Efficacy of Regional Intravenous (RI) Versus Intramuscular (IM) Delivery of rAAV1 and rAAV8 to Nonhuman Primate Skeletal Muscle

(2008) Alice Toromanoff et al.   MOLECULAR 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