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49. Minimum Effective Dose of Liver-Targeted Gene Therapy for Pompe Disease Reduced 10-Fold from Prior Estimates
Han, Sang-oh, Amson, Benjamin, Busch, Korie, Ronzitti, Giuseppe, Li, Songtao, Mingozzi, Federico, Koeberl, Dwight D.Volume:
24
Language:
english
Journal:
Molecular Therapy
DOI:
10.1016/s1525-0016(16)32858-1
Date:
May, 2016
File:
PDF, 844 KB
english, 2016