检索结果 - Dwight D. Koeberl

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  1. 1
    Gene therapy for glycogen storage diseases

    Gene therapy for glycogen storage diseases 由 Priya S. Kishnani, Baodong Sun, Dwight D. Koeberl

    出版 2019
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  2. 2
    Glycogen storage disease types I and II: Treatment updates

    Glycogen storage disease types I and II: Treatment updates 由 Dwight D. Koeberl, Priya S. Kishnani, Yan‐Chu Chen

    出版 2007
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  3. 3
    The Role of Hepatocyte Hemojuvelin in the Regulation of Bone Morphogenic Protein-6 and Hepcidin Expression in Vivo

    The Role of Hepatocyte Hemojuvelin in the Regulation of Bone Morphogenic Protein-6 and Hepcidin Expression in Vivo 由 An‐Sheng Zhang, Junwei Gao, Dwight D. Koeberl, Caroline Enns

    出版 2010
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  4. 4
    Fasting alleviates metabolic alterations in mice with propionyl-CoA carboxylase deficiency due to Pcca mutation

    Fasting alleviates metabolic alterations in mice with propionyl-CoA carboxylase deficiency due to Pcca mutation 由 Wentao He, Hannah Marchuk, Dwight D. Koeberl, Takhar Kasumov, Xiaoxin Chen, Guofang Zhang

    出版 2024
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  5. 5
    Enhanced Response to Enzyme Replacement Therapy in Pompe Disease after the Induction of Immune Tolerance

    Enhanced Response to Enzyme Replacement Therapy in Pompe Disease after the Induction of Immune Tolerance 由 Baodong Sun, Andrew Bird, Sarah P. Young, Priya S. Kishnani, Yuqi Chen, Dwight D. Koeberl

    出版 2007
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  6. 6
    Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter

    Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter 由 Baodong Sun, Haoyue Zhang, Luis M. Franco, T. T. Brown, Andrew Bird, Ayn Schneider, Dwight D. Koeberl

    出版 2005
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  7. 7
    Persistent expression of human clotting factor IX from mouse liver after intravenous injection of adeno-associated virus vectors

    Persistent expression of human clotting factor IX from mouse liver after intravenous injection of adeno-associated virus vectors 由 Dwight D. Koeberl, Ian E. Alexander, Christine L. Halbert, David W. Russell, A. Dusty Miller

    出版 1997
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  8. 8
    Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction

    Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction 由 Sang-oh Han, Giuseppe Ronzitti, Benjamin Arnson, Christian Leborgne, Songtao Li, Federico Mingozzi, Dwight D. Koeberl

    出版 2017
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  9. 9
    Efficacy of an Adeno-associated Virus 8-Pseudotyped Vector in Glycogen Storage Disease Type II

    Efficacy of an Adeno-associated Virus 8-Pseudotyped Vector in Glycogen Storage Disease Type II 由 Baodong Sun, Haoyue Zhang, Luis M. Franco, Sarah P. Young, Ayn Schneider, Andrew Bird, Andrea Amalfitano, Y.-T. Chen, Dwight D. Koeberl

    出版 2004
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  10. 10
    Hepatocyte-targeted HFE and TFR2 control hepcidin expression in mice

    Hepatocyte-targeted HFE and TFR2 control hepcidin expression in mice 由 Junwei Gao, Juxing Chen, Ivana De Domenico, David M. Koeller, Cary O. Harding, Robert E. Fleming, Dwight D. Koeberl, Caroline Enns

    出版 2010
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  11. 11
    Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal muscle

    Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal muscle 由 Dwight D. Koeberl, Xiaoyan Luo, Baodong Sun, Alison McVie‐Wylie, Jian Dai, Songtao Li, Suhrad G. Banugaria, Y.-T. Chen, Deeksha Bali

    出版 2011
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  12. 12
    Immunodominant Liver-Specific Expression Suppresses Transgene-Directed Immune Responses in Murine Pompe Disease

    Immunodominant Liver-Specific Expression Suppresses Transgene-Directed Immune Responses in Murine Pompe Disease 由 Ping Zhang, Baodong Sun, Takuya Osada, Ramona M. Rodriguiz, Xiao Yang, Xiaoyan Luo, Alex R. Kemper, Timothy M. Clay, Dwight D. Koeberl

    出版 2012
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  13. 13
    A Liver-Specific Thyromimetic, VK2809, Decreases Hepatosteatosis in Glycogen Storage Disease Type Ia

    A Liver-Specific Thyromimetic, VK2809, Decreases Hepatosteatosis in Glycogen Storage Disease Type Ia 由 Jin Zhou, Lauren R. Waskowicz, Andrea Lim, Xiao-Hui Liao, Brian Lian, Hiroko Masamune, Samuel Refetoff, Brian Tran, Dwight D. Koeberl, Paul M. Yen

    出版 2019
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  14. 14
    Evasion of Immune Responses to Introduced Human Acid α-Glucosidase by Liver-Restricted Expression in Glycogen Storage Disease Type II

    Evasion of Immune Responses to Introduced Human Acid α-Glucosidase by Liver-Restricted Expression in Glycogen Storage Disease Type II 由 Luis M. Franco, Baodong Sun, Xiaoyi Yang, Andrew Bird, Haoyue Zhang, Ayn Schneider, T. T. Brown, Sarah P. Young, Timothy M. Clay, Andrea Amalfitano, Y.T. Chen, Dwight D. Koeberl

    出版 2005
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  15. 15
    Hepatic mitochondrial dysfunction is a feature of Glycogen Storage Disease Type Ia (GSDIa)

    Hepatic mitochondrial dysfunction is a feature of Glycogen Storage Disease Type Ia (GSDIa) 由 Benjamin L. Farah, Rohit A. Sinha, Yajun Wu, Brijesh Kumar Singh, Andrea Lim, Masahiro Hirayama, Dustin J. Landau, Boon‐Huat Bay, Dwight D. Koeberl, Paul M. Yen

    出版 2017
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  16. 16
    Interim analyses of a first-in-human phase 1/2 mRNA trial for propionic acidaemia

    Interim analyses of a first-in-human phase 1/2 mRNA trial for propionic acidaemia 由 Dwight D. Koeberl, Andreas Schulze, Neal Sondheimer, Gerald S. Lipshutz, Tarekegn Geberhiwot, Lerong Li, Rajnish Saini, Junxiang Luo, Vanja Sikirica, Ling Jin, M. Liang, Mary Leuchars, Stephanie Grünewald

    出版 2024
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  17. 17
    Immunomodulatory Gene Therapy Prevents Antibody Formation and Lethal Hypersensitivity Reactions in Murine Pompe Disease

    Immunomodulatory Gene Therapy Prevents Antibody Formation and Lethal Hypersensitivity Reactions in Murine Pompe Disease 由 Baodong Sun, Michael D. Kulis, Sarah P. Young, Amy Hobeika, Songtao Li, Andrew Bird, Haoyue Zhang, Y Li, Timothy M. Clay, Wesley Burks, Priya S. Kishnani, Dwight D. Koeberl

    出版 2009
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  18. 18
    Correction of Multiple Striated Muscles in Murine Pompe Disease Through Adeno-associated Virus–mediated Gene Therapy

    Correction of Multiple Striated Muscles in Murine Pompe Disease Through Adeno-associated Virus–mediated Gene Therapy 由 Baodong Sun, Sarah P. Young, Ping Li, Chunhui Di, T. T. Brown, Maja Z. Salva, Songtao Li, Andrew Bird, Zhen Yan, Richard L. Auten, Stephen D. Hauschka, Dwight D. Koeberl

    出版 2008
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  19. 19
    Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants

    Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants 由 Priya S. Kishnani, Paula Goldenberg, Stephanie DeArmey, James H. Heller, Danny Benjamin, Sarah P. Young, Deeksha Bali, Sue Ann Smith, Jennifer S. Li, Hanna Mandel, Dwight D. Koeberl, Amy S. Rosenberg, Y-T Chen

    出版 2009
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  20. 20
    Chromosomal and genetic alterations in human hepatocellular adenomas associated with type Ia glycogen storage disease

    Chromosomal and genetic alterations in human hepatocellular adenomas associated with type Ia glycogen storage disease 由 Priya S. Kishnani, Tzu‐Po Chuang, Deeksha Bali, Dwight D. Koeberl, Stephanie Austin, David A. Weinstein, Elaine Murphy, Ying‐Ting Chen, Keri Boyette, Chu‐Hao Liu, Yuan-Tsong Chen, Ling‐Hui Li

    出版 2009
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