In Vivo Zinc Finger Nuclease-mediated Targeted Integration of a Glucose-6-phosphatase Transgene Promotes Survival in Mice With Glycogen Storage Disease Type IA

par Landau, Dustin J, Brooks, Elizabeth Drake, Perez-Pinera, Pablo, Amarasekara, Hiruni, Mefferd, Adam, Li, Songtao, Bird, Andrew, Gersbach, Charles A, Koeberl, Dwight D
Publié dans Mol Ther (2016)

Pathogenesis of growth failure and partial reversal with gene therapy in murine and canine Glycogen Storage Disease type Ia

par Brooks, Elizabeth Drake, Little, Dianne, Arumugam, Ramamani, Sun, Baodong, Curtis, Sarah, DeMaster, Amanda, Maranzano, Michael, Jackson, Mark W., Kishnani, Priya, Freemark, Michael S., Koeberl, Dwight D.
Publié 2013