Liver-directed adeno-associated virus serotype 8 gene transfer rescues a lethal murine model of citrullinemia type 1

Liver transplantation for urea cycle disorders in pediatric patients: A single-center experience

(2013) Irene K. Kim et al.   PEDIATRIC TRANSPLANTATION

Pre-clinical efficacy and dosing of an AAV8 vector expressing human methylmalonyl-CoA mutase in a murine model of methylmalonic acidemia (MMA)

(2012) Randy J. Chandler et al.   MOLECULAR GENETICS AND METABOLISM

Preclinical evaluation of a clinical candidate AAV8 vector for ornithine transcarbamylase (OTC) deficiency reveals functional enzyme from each persisting vector genome

(2011) Lili Wang et al.   MOLECULAR GENETICS AND METABOLISM

Two Hypomorphic Alleles of Mouse Ass1 as a New Animal Model of Citrullinemia Type I and Other Hyperammonemic Syndromes

(2010) Carlos J. Perez et al.   AMERICAN JOURNAL OF PATHOLOGY

Adeno-Associated Virus Serotype 8 Gene Transfer Rescues a Neonatal Lethal Murine Model of Propionic Acidemia

(2010) Randy J. Chandler et al.   HUMAN GENE THERAPY

AAV2/8-mediated Correction of OTC Deficiency Is Robust in Adult but Not Neonatal Spfash Mice

(2009) Sharon C Cunningham et al.   MOLECULAR THERAPY