Koeberl, Dwight D.

Immunodominant liver-specific expression suppresses transgene-directed immune responses in murine pompe disease

Zhang, P., Sun, B., Osada, T., Rodriguiz, R., Yang, X. Y., Luo, X., Kemper, A. R., Clay, T. M., & Koeberl, D. D. (2012) · Human Gene Therapy

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

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Low-dose liver-targeted gene therapy for pompe disease enhances therapeutic efficacy of ERT via immune tolerance induction

Han, S.-O., Ronzitti, G., Arnson, B., Leborgne, C., Li, S., Mingozzi, F., & Koeberl, D. (2017) · Molecular Therapy - Methods & Clinical Development

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

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Person Ability Scores as an Alternative to Norm-Referenced Scores as Outcome Measures in Studies of Neurodevelopmental Disorders

Farmer, C. A., Kaat, A. J., Thurm, A., Anselm, I., Akshoomoff, N., Bennett, A., Berry, L., Bruchey, A., Barshop, B. A., Berry-Kravis, E., et al. (2020) · American Journal on Intellectual and Developmental Disabilities

Psychometric Methodologies and TestingAutism Spectrum Disorder Research

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A Liver-Specific Thyromimetic, VK2809, Decreases Hepatosteatosis in Glycogen Storage Disease Type Ia

Zhou, J., Waskowicz, L. R., Lim, A., Liao, X.-H., Lian, B., Masamune, H., Refetoff, S., Tran, B., Koeberl, D. D., & Yen, P. M. (2019) · Thyroid®

Glycogen Storage Diseases and MyoclonusMetabolism and Genetic Disorders

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Correction of Biochemical Abnormalities and Improved Muscle Function in a Phase I/II Clinical Trial of Clenbuterol in Pompe Disease

Koeberl, D. D., Case, L. E., Smith, E. C., Walters, C., Han, S.-O., Li, Y., Chen, W., Hornik, C. P., Huffman, K. M., Kraus, W. E., et al. (2018) · Molecular Therapy

Lysosomal Storage Disorders ResearchGlycogen Storage Diseases and Myoclonus

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