49. Minimum Effective Dose of Liver-Targeted Gene Therapy for Pompe Disease Reduced 10-Fold from Prior Estimates
Sang-oh Han(Duke University), Dwight D. Koeberl(Duke Medical Center), Benjamin Amson(Duke Medical Center), Songtao Li(Zhejiang Chinese Medical University), Federico Mingozzi(Spark Therapeutics (United States)), Giuseppe Ronzitti(Inserm), Korie Busch(Duke Medical Center)
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