Rescue administration of a helper-dependent adenovirus vector with long-term efficacy in dogs with glycogen storage disease type Ia

Neonatal helper-dependent adenoviral vector gene therapy mediates correction of hemophilia A and tolerance to human factor VIII

(2011) Chuhong Hu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Adeno-Associated Virus-Mediated Correction of a Canine Model of Glycogen Storage Disease Type Ia

(2010) David A. Weinstein et al.   HUMAN GENE THERAPY

Complete Normalization of Hepatic G6PC Deficiency in Murine Glycogen Storage Disease Type Ia Using Gene Therapy

(2010) Wai Han Yiu et al.   MOLECULAR THERAPY

Normoglycemia alone is insufficient to prevent long-term complications of hepatocellular adenoma in glycogen storage disease type Ib mice

(2009) Wai Han Yiu et al.   JOURNAL OF HEPATOLOGY

Progress and prospects: gene therapy for genetic diseases with helper-dependent adenoviral vectors

(2008) N Brunetti-Pierri et al.   GENE THERAPY

AAV Vector-mediated Reversal of Hypoglycemia in Canine and Murine Glycogen Storage Disease Type Ia

(2008) Dwight D Koeberl et al.   MOLECULAR THERAPY

Efficient, Long-term Hepatic Gene Transfer Using Clinically Relevant HDAd Doses by Balloon Occlusion Catheter Delivery in Nonhuman Primates

(2008) Nicola Brunetti-Pierri et al.   MOLECULAR THERAPY