Ontology highlight
ABSTRACT:
SUBMITTER: Gau CL
PROVIDER: S-EPMC2835205 | biostudies-literature | 2009 Jul
REPOSITORIES: biostudies-literature
Gau Chia-Ling CL Rosenblatt Robin A RA Cerullo Vincenzo V Lay Fides D FD Dow Adrienne C AC Livesay Justin J Brunetti-Pierri Nicola N Lee Brendan B Cederbaum Stephen D SD Grody Wayne W WW Lipshutz Gerald S GS
Molecular therapy : the journal of the American Society of Gene Therapy 20090414 7
Neonatal gene therapy has the potential to ameliorate abnormalities before disease onset. Our gene knockout of arginase I (AI) deficiency is characterized by increasing hyperammonemia, neurological deterioration, and early death. We constructed a helper-dependent adenoviral vector (HDV) carrying AI and examined for correction of this defect. Neonates were administered 5 x 10(9) viral particles/g and analyzed for survival, arginase activity, and ammonia and amino acids levels. The life expectancy ...[more]