Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction

Sang-oh Han(Duke University), Dwight D. Koeberl(Duke Medical Center), Benjamin Arnson(Duke University), Songtao Li(Zhejiang Chinese Medical University), Federico Mingozzi(Spark Therapeutics (United States)), Christian Leborgne(Inserm), Giuseppe Ronzitti(Inserm)
Molecular Therapy — Methods & Clinical Development
May 1, 2019
10.1016/j.omtm.2019.04.005
Cited by 6

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