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Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enha..:

Han, Sang-oh ; Ronzitti, Giuseppe ; Arnson, Benjamin...
Molecular Therapy - Methods & Clinical Development.  4 (2017)  - p. 126-136 , 2017
Link: https://doi.org/10.1016/j.omtm.2016.12.010