Search Results - Cary O Harding

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  1. 1

    Prospects for Cell-Directed Curative Therapy of Phenylketonuria (PKU) by Cary O. Harding

    Published in Molecular Frontiers Journal (2019-12-01)
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  2. 2

    Letter to the editor by Cary O. Harding, Michael Martinez

    Published in HGG Advances (2024-07-01)
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  3. 3

    A Sensitive Assay System To Test Antisense Oligonucleotides for Splice Suppression Therapy in the Mouse Liver by Lorena Gallego-Villar, Hiu Man Viecelli, Belén Pérez, Cary O Harding, Magdalena Ugarte, Beat Thöny, Lourdes R Desviat

    Published in Molecular Therapy: Nucleic Acids (2014-01-01)
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  4. 4

    Low-Dose Gene Therapy for Murine PKU Using Episomal Naked DNA Vectors Expressing PAH from Its Endogenous Liver Promoter by Hiu Man Grisch-Chan, Andrea Schlegel, Tanja Scherer, Gabriella Allegri, Raphael Heidelberger, Panagiota Tsikrika, Marco Schmeer, Martin Schleef, Cary O. Harding, Johannes Häberle, Beat Thöny

    Published in Molecular Therapy: Nucleic Acids (2017-06-01)
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  5. 5

    Intelligence quotient scores among early-treated phenylketonuria patients: results from a systematic literature review by Fiona O’Sullivan, Ioannis Tomazos, Francjan J. van Spronsen, Shelagh M. Szabo, Maanasa Venkataraman, Lavanya Huria, Neil Smith, Lachlan Molony, Kim Ingalls, Kathleen Somera-Molina, Rongrong Zhang, Cary O. Harding

    Published in Orphanet Journal of Rare Diseases (2025-06-01)
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  6. 6

    A G1528C Hadha knock-in mouse model recapitulates aspects of human clinical phenotypes for long-chain 3-hydroxyacyl-CoA dehydrogenase deficiency by Garen Gaston, Shannon Babcock, Renee Ryals, Gabriela Elizondo, Tiffany DeVine, Dahlia Wafai, William Packwood, Sarah Holden, Jacob Raber, Jonathan R. Lindner, Mark E. Pennesi, Cary O. Harding, Melanie B. Gillingham

    Published in Communications Biology (2023-08-01)
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  7. 7

    Pegvaliase for the treatment of phenylketonuria: Final results of a long-term phase 3 clinical trial program by Cary O. Harding, Nicola Longo, Hope Northrup, Stephanie Sacharow, Rani Singh, Janet A. Thomas, Jerry Vockley, Roberto T. Zori, Kaleigh Bulloch Whitehall, Joshua Lilienstein, Kristin Lindstrom, Drew G. Levy, Shaun Jones, Barbara K. Burton

    Published in Molecular Genetics and Metabolism Reports (2024-06-01)
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  8. 8

    Pharmacokinetic, pharmacodynamic, and immunogenic rationale for optimal dosing of pegvaliase, a PEGylated bacterial enzyme, in adult patients with phenylketonuria by Yulan Qi, Gina Patel, Joshua Henshaw, Soumi Gupta, Joy Olbertz, Kevin Larimore, Cary O. Harding, Markus Merilainen, Roberto Zori, Nicola Longo, Barbara K. Burton, Mingjin Li, Zhonghua Gu, Stephen J. Zoog, Haoling H. Weng, Becky Schweighardt

    Published in Clinical and Translational Science (2021-09-01)
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  9. 9

    Meta-analysis of bone mineral density in adults with phenylketonuria by Júlio C. Rocha, Álvaro Hermida, Cheryl J. Jones, Yunchou Wu, Gillian E. Clague, Sarah Rose, Kaleigh B. Whitehall, Kirsten K. Ahring, André L. S. Pessoa, Cary O. Harding, Fran Rohr, Anita Inwood, Nicola Longo, Ania C. Muntau, Serap Sivri, François Maillot

    Published in Orphanet Journal of Rare Diseases (2024-09-01)
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  10. 10

    Systematic literature review of the somatic comorbidities experienced by adults with phenylketonuria by Kaleigh B. Whitehall, Sarah Rose, Gillian E. Clague, Kirsten K. Ahring, Deborah A. Bilder, Cary O. Harding, Álvaro Hermida, Anita Inwood, Nicola Longo, François Maillot, Ania C. Muntau, André L. S. Pessoa, Júlio C. Rocha, Fran Rohr, Serap Sivri, Jack Said, Sheun Oshinbolu, Gillian C. Sibbring

    Published in Orphanet Journal of Rare Diseases (2024-08-01)
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