title: Monitoring the correction of glycogen storage disease type 1a in a mouse model using [(18)F]FDG and a dedicated animal scanner.
abstract: Monitoring gene therapy of glycogen storage disease type 1a in a mouse model was achieved using [(18)F]FDG and a dedicated animal scanner. The G6Pase knockout (KO) mice were compared to the same mice after infusion with a recombinant adenovirus containing the murine G6Pase gene (Ad-mG6Pase). Serial images of the same mouse before and after therapy were obtained and compared with wild-type (WT) mice of the same strain to determine the uptake and retention of [(18)F]FDG in the liver. Image data were acquired from heart, blood pool and liver for twenty minutes after injection of [(18)F]FDG. The retention of [(18)F]FDG was lower for the WT mice compared to the KO mice. The mice treated with adenovirus-mediated gene therapy had retention similar to that found in age-matched WT mice. These studies show that FDG can be used to monitor the G6Pase concentration in liver of WT mice as compared to G6Pase KO mice. In these mice, gene therapy returned the liver function to that found in age matched WT controls as measured by the FDG kinetics in the liver compared to that found in age matched wild type controls.
authors.affiliation: National Institute of Child Health and Development, 20892, Bethesda MD, USA
authors.forename: A
authors.initials: A
authors.lastname: Zingone
authors.forename: J
authors.initials: J
authors.lastname: Seidel
authors.forename: L
authors.initials: L
authors.lastname: Aloj
authors.forename: C
authors.initials: C
authors.lastname: Caraco
authors.forename: J J
authors.initials: JJ
authors.lastname: Vaquero
authors.forename: E M
authors.initials: EM
authors.lastname: Jagoda
authors.forename: J Y
authors.initials: JY
authors.lastname: Chou
authors.forename: M V
authors.initials: MV
authors.lastname: Green
authors.forename: W C
authors.initials: WC
authors.lastname: Eckelman
mesh_terms: D000818:Animals; D004195:Disease Models, Animal; D019788:Fluorodeoxyglucose F18 / Q000378:metabolism*; D015316:Genetic Therapy; D005947:Glucose / Q000378:metabolism; D005952:Glucose-6-Phosphatase / Q000235:genetics*; D005953:Glycogen Storage Disease Type I / Q000000981:diagnostic imaging* / Q000235:genetics / Q000378:metabolism / Q000628:therapy*; D006801:Humans; D051379:Mice; D008807:Mice, Inbred BALB C; D018345:Mice, Knockout; D019275:Radiopharmaceuticals / Q000378:metabolism; D014055:Tomography, Emission-Computed
chemical_list: D019275:Radiopharmaceuticals; D019788:Fluorodeoxyglucose F18; D005952:Glucose-6-Phosphatase; D005947:Glucose