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In Vivo Zinc Finger Nuclease-Mediated Targeted Integration of a Glucose-6-Phosphatase Transgene Promotes Survival in Mice with Glycogen Storage Disease Type IA
Landau, Dustin J., Brooks, Elizabeth Drake, Perez-Pinera, Pablo, Amarasekara, Hiruni, Mefferd, Adam, Li, Songtao, Bird, Andrew, Gersbach, Charles A., Koeberl, Dwight D.Language:
english
Journal:
Molecular Therapy
DOI:
10.1038/mt.2016.35
Date:
February, 2016
File:
PDF, 7.73 MB
english, 2016