Hasil Pencarian - Dwight D. Koeberl

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  1. 1
    Complex III deficiency due to an in-frame MT-CYB deletion presenting as ketotic hypoglycemia and lactic acidosis

    Complex III deficiency due to an in-frame MT-CYB deletion presenting as ketotic hypoglycemia and lactic acidosis oleh Mari Mori, Jennifer Goldstein, Sarah P. Young, Edward H. Bossen, John Shoffner, Dwight D. Koeberl

    Diterbitkan 2015-09-01
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  2. 2
    Assessment of toxicity and biodistribution of recombinant AAV8 vector–mediated immunomodulatory gene therapy in mice with Pompe disease

    Assessment of toxicity and biodistribution of recombinant AAV8 vector–mediated immunomodulatory gene therapy in mice with Pompe disease oleh Gensheng Wang, Sarah P Young, Deeksha Bali, Julie Hutt, Songtao Li, Janet Benson, Dwight D Koeberl

    Diterbitkan 2014-01-01
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  3. 3
    Corrigendum to âAssessment of toxicity and biodistribution of recombinant AAV8 vectorâmediated immunomodulatory gene therapy in mice with Pompe diseaseâ

    Corrigendum to âAssessment of toxicity and biodistribution of recombinant AAV8 vectorâmediated immunomodulatory gene therapy in mice with Pompe diseaseâ oleh Gensheng Wang, Sarah P Young, Deeksha Bali, Julie Hutt, Songtao Li, Janet Benson, Dwight D Koeberl

    Diterbitkan 2015-01-01
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  4. 4
    Bezafibrate Enhances AAV Vector-Mediated Genome Editing in Glycogen Storage Disease Type Ia

    Bezafibrate Enhances AAV Vector-Mediated Genome Editing in Glycogen Storage Disease Type Ia oleh Hye-Ri Kang, Lauren Waskowicz, Andrea M. Seifts, Dustin J. Landau, Sarah P. Young, Dwight D. Koeberl

    Diterbitkan 2019-06-01
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  5. 5
    Characterization of a canine model of glycogen storage disease type IIIa

    Characterization of a canine model of glycogen storage disease type IIIa oleh Haiqing Yi, Beth L. Thurberg, Sarah Curtis, Stephanie Austin, John Fyfe, Dwight D. Koeberl, Priya S. Kishnani, Baodong Sun

    Diterbitkan 2012-11-01
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  6. 6
    Screening data from 19 patients with late‐onset Pompe disease for a phase I clinical trial of AAV8 vector‐mediated gene therapy

    Screening data from 19 patients with late‐onset Pompe disease for a phase I clinical trial of AAV8 vector‐mediated gene therapy oleh William B. Hannah, Laura E. Case, Edward C. Smith, Crista Walters, Deeksha Bali, Priya S. Kishnani, Dwight D. Koeberl

    Diterbitkan 2023-09-01
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  7. 7
    Genome editing using Staphylococcus aureus Cas9 in a canine model of glycogen storage disease Ia

    Genome editing using Staphylococcus aureus Cas9 in a canine model of glycogen storage disease Ia oleh Benjamin Arnson, Hye Ri Kang, Elizabeth D. Brooks, Dorothy Gheorghiu, Ekaterina Ilich, David Courtney, Jeffrey I. Everitt, Bryan R. Cullen, Dwight D. Koeberl

    Diterbitkan 2023-06-01
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  8. 8
    Albuterol as an adjunctive treatment to enzyme replacement therapy in infantile-onset Pompe disease

    Albuterol as an adjunctive treatment to enzyme replacement therapy in infantile-onset Pompe disease oleh Yin-Hsiu Chien, Wuh-Liang Hwu, Ni-Chung Lee, Fuu-Jen Tsai, Dwight D. Koeberl, Wen-Hui Tsai, Pao-Chin Chiu, Chaw-Liang Chang

    Diterbitkan 2017-06-01
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  9. 9
    Three-dimensional tissue-engineered human skeletal muscle model of Pompe disease

    Three-dimensional tissue-engineered human skeletal muscle model of Pompe disease oleh Jason Wang, Chris J. Zhou, Alastair Khodabukus, Sabrina Tran, Sang-Oh Han, Aaron L. Carlson, Lauran Madden, Priya S. Kishnani, Dwight D. Koeberl, Nenad Bursac

    Diterbitkan 2021-05-01
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  10. 10
    Comparisons of Infant and Adult Mice Reveal Age Effects for Liver Depot Gene Therapy in Pompe Disease

    Comparisons of Infant and Adult Mice Reveal Age Effects for Liver Depot Gene Therapy in Pompe Disease oleh Sang-oh Han, Songtao Li, Angela McCall, Benjamin Arnson, Jeffrey I. Everitt, Haoyue Zhang, Sarah P. Young, Mai K. ElMallah, Dwight D. Koeberl

    Diterbitkan 2020-06-01
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  11. 11
    Itavastatin and resveratrol increase triosephosphate isomerase protein in a newly identified variant of TPI deficiency

    Itavastatin and resveratrol increase triosephosphate isomerase protein in a newly identified variant of TPI deficiency oleh Andrew P. VanDemark, Stacy L. Hrizo, Samantha L. Eicher, Jules Kowalski, Tracey D. Myers, Megan R. Pfeifer, Kacie N. Riley, Dwight D. Koeberl, Michael J. Palladino

    Diterbitkan 2022-05-01
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  12. 12
    Pathogenesis of Hepatic Tumors following Gene Therapy in Murine and Canine Models of Glycogen Storage Disease

    Pathogenesis of Hepatic Tumors following Gene Therapy in Murine and Canine Models of Glycogen Storage Disease oleh Hye-Ri Kang, Monika Gjorgjieva, Stephanie N. Smith, Elizabeth D. Brooks, Zelin Chen, Shawn M. Burgess, Randy J. Chandler, Lauren R. Waskowicz, Kylie M. Grady, Songtao Li, Gilles Mithieux, Charles P. Venditti, Fabienne Rajas, Dwight D. Koeberl

    Diterbitkan 2019-12-01
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