Search Results - Dwight D. Koeberl
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Gene therapy for glycogen storage diseases by Priya S. Kishnani, Baodong Sun, Dwight D. Koeberl
Published 2019Revisão -
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Glycogen storage disease types I and II: Treatment updates by Dwight D. Koeberl, Priya S. Kishnani, Yan‐Chu Chen
Published 2007Revisão -
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Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal muscle by Dwight D. Koeberl, Xiaoyan Luo, Baodong Sun, Alison McVie‐Wylie, Jian Dai, Songtao Li, Suhrad G. Banugaria, Y.-T. Chen, Deeksha Bali
Published 2011Artigo -
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Evasion of Immune Responses to Introduced Human Acid α-Glucosidase by Liver-Restricted Expression in Glycogen Storage Disease Type II by 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
Published 2005Artigo -
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Hepatic mitochondrial dysfunction is a feature of Glycogen Storage Disease Type Ia (GSDIa) by 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
Published 2017Artigo -
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Interim analyses of a first-in-human phase 1/2 mRNA trial for propionic acidaemia by 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
Published 2024Artigo -
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Immunomodulatory Gene Therapy Prevents Antibody Formation and Lethal Hypersensitivity Reactions in Murine Pompe Disease by 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
Published 2009Artigo -
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Correction of Multiple Striated Muscles in Murine Pompe Disease Through Adeno-associated Virus–mediated Gene Therapy by 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
Published 2008Artigo -
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Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants by 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
Published 2009Artigo -
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Chromosomal and genetic alterations in human hepatocellular adenomas associated with type Ia glycogen storage disease by 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
Published 2009Artigo
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Medicine
Biology
Internal medicine
Biochemistry
Endocrinology
Gene
Disease
Genetic enhancement
Enzyme replacement therapy
Glycogen
Glycogen storage disease
Glycogen storage disease type II
Immunology
Recombinant DNA
Virology
Genetics
Vector (molecular biology)
Adeno-associated virus
Chemistry
Immune system
Antibody
Viral vector
Glycogen storage disease type I
Hepatocyte
In vitro
Molecular biology
Pathology
Skeletal muscle
Archaeology
Clinical trial