In Vivo Zinc Finger Nuclease-mediated Targeted Integration of a Glucose-6-phosphatase Transgene Promotes Survival in Mice With Glycogen Storage Disease Type IA
Molecular Therapy2016Vol. 24(4), pp. 697–706
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Dustin J. Landau, Elizabeth D. Brooks, Pablo Pérez‐Piñera, Hiruni S. Amarasekara, Adam Mefferd, Songtao Li, Andrew Bird, Charles A. Gersbach, Dwight D. Koeberl
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