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Human Gene Therapy|September 30, 2015
Synergistic Efficacy from Gene Therapy with Coreceptor Blockade and a β2-Agonist in Murine Pompe DiseaseSang-oh Han, Songtao Li, Andrew Bird, et al.Human Gene Therapy|February 1, 2022
Minimum Effective Dose to Achieve Biochemical Correction with Adeno-Associated Virus Vector-Mediated Gene Therapy in Mice with Pompe DiseaseSang-Oh Han, Dorothy Gheorghiu, Songtao Li, et al.Communications Biology|May 29, 2024
Fasting alleviates metabolic alterations in mice with propionyl-CoA carboxylase deficiency due to Pcca mutationWentao He, Hannah Marchuk, Dwight Koeberl, et al.Journal of Clinical and Translational Science|May 19, 2025
Developing a public-facing tool to monitor inclusion of special populations in clinical researchWendy K K Lam, James Goodrich, Sydney Sullivan, et al.Molecular Therapy. Methods & Clinical Development|March 28, 2017
Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance InductionSang-Oh Han, Giuseppe Ronzitti, Benjamin Arnson, et al.Molecular Therapy. Methods & Clinical Development|May 11, 2019
Erratum: Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance InductionSang-Oh Han, Giuseppe Ronzitti, Benjamin Arnson, et al.American Journal of Medical Genetics. Part A|April 29, 2024
The interplay of psychosis and non-compliance with fatal outcome in an adult with MSUDNadia Falah, Surekha Pendyal, Cina Sasannejad, et al.Molecular Genetics and Metabolism Reports|March 12, 2024
Critical sample collection delayed? Urine organic acid analysis can still save the day! A new case of HMG-CoA synthase deficiencyMonika Williams, Iskren Menkovic, Pamela Reitnauer, et al.Human Gene Therapy|January 27, 2022
Efficacious Androgen Hormone Administration in Combination with Adeno-Associated Virus Vector-Mediated Gene Therapy in Female Mice with Pompe DiseaseSang-Oh Han, Dorothy Gheorghiu, Alex Chang, et al.IEEE Transactions on Ultrasonics, Ferroelectrics, and Frequency Control|February 15, 2013
Enhanced gene expression of systemically administered plasmid DNA in the liver with therapeutic ultrasound and microbubblesBalasundar I Raju, Evgeniy Leyvi, Ralf Seip, et al.Pageof 3