Publications by authors named "Dwight D Koeberl"

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Developing therapeutics for rare cardiovascular diseases.
Joseph B Lerman, Dwight D Koeberl, Shilpi Epstein, Lothar Roessig, Rodica Stan

Am Heart J· February 2026

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Timely intervention in HMG-CoA Lyase deficiency: The role of newborn screening, metabolic management, and genomic sequencing.
Iskren Menkovic, Neelam Makhijani, Ludmila Francescatto, Surekha Pendyal, Christine Stanley, Dwight D Koeberl

Mol Genet Metab Rep· December 2025

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Longitudinal Characterization of Males With X-Linked Creatine Transporter Deficiency: Final Results of a Multiyear Observational Study.
Judith S Miller, Cristan Farmer, Susan Blair, Simona Bianconi, Natacha Akshoomoff, Dwight D Koeberl

Pediatr Neurol· October 2025

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Efficacious genome editing in infant mice with glycogen storage disease type Ia.
Benjamin Arnson, Ekaterina Ilich, Troy von Beck, Songtao Li, Elizabeth D Brooks, Dwight D Koeberl

JCI Insight· September 2025

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Staying on target in gene and cell therapy.
Rory Bricker-Anthony, Dwight D Koeberl, Gerald S Lipshutz, Fabiana Perna

Mol Ther· September 2024

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Gene therapy for glycogen storage diseases.
Dwight D Koeberl, Rebecca L Koch, Jeong-A Lim, Elizabeth D Brooks, Benjamin D Arnson

J Inherit Metab Dis· January 2024

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

JIMD Rep· September 2023

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Successful AAV8 readministration: Suppression of capsid-specific neutralizing antibodies by a combination treatment of bortezomib and CD20 mAb in a mouse model of Pompe disease.
Su Jin Choi, John S Yi, Jeong-A Lim, Thomas F Tedder, Dwight D Koeberl

J Gene Med· August 2023

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Phase I study of liver depot gene therapy in late-onset Pompe disease.
Edward C Smith, Sam Hopkins, Laura E Case, Ming Xu, Crista Walters, Dwight D Koeberl

Mol Ther· July 2023

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Genome editing using Cas9 in a canine model of glycogen storage disease Ia.
Benjamin Arnson, Hye Ri Kang, Elizabeth D Brooks, Dorothy Gheorghiu, Ekaterina Ilich, Dwight D Koeberl

Mol Ther Methods Clin Dev· June 2023

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Contributions from medical geneticists in clinical trials of genetic therapies: A points to consider statement of the American College of Medical Genetics and Genomics (ACMG).
Loren D M Peña, Lindsay C Burrage, Gregory M Enns, Edward D Esplin, Cary Harding, Dwight D Koeberl

Genet Med· June 2023

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Clinical, biochemical and molecular characterization of 12 patients with pyruvate carboxylase deficiency treated with triheptanoin.
M Laura Duque Lasio, Angela C Leshinski, Nicole H Ducich, Leigh Anne Flore, April Lehman, Dwight D Koeberl

Mol Genet Metab· June 2023

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Clenbuterol Treatment Is Safe and Associated With Slowed Disease Progression in a Small Open-Label Trial in Patients With Amyotrophic Lateral Sclerosis.
Xiaoyan Li, Dwight D Koeberl, Michael W Lutz, Richard Bedlack

J Clin Neuromuscul Dis· June 2023

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Moving away from one disease at a time: Screening, trial design, and regulatory implications of novel platform technologies.
Julie Lekstrom-Himes, P J Brooks, Dwight D Koeberl, Amy Brower, Aaron Goldenberg

Am J Med Genet C Semin Med Genet· March 2023

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Itavastatin and resveratrol increase triosephosphate isomerase protein in a newly identified variant of TPI deficiency.
Andrew P VanDemark, Stacy L Hrizo, Samantha L Eicher, Jules Kowalski, Tracey D Myers, Dwight D Koeberl

Dis Model Mech· May 2022

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Three-dimensional tissue-engineered human skeletal muscle model of Pompe disease.
Jason Wang, Chris J Zhou, Alastair Khodabukus, Sabrina Tran, Sang-Oh Han, Dwight D Koeberl

Commun Biol· May 2021

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Adeno-Associated Virus-Mediated Gene Therapy in the Mashlool, , Mouse Model of Alternating Hemiplegia of Childhood.
Arsen S Hunanyan, Boris Kantor, Ram S Puranam, Courtney Elliott, Angela McCall, Dwight D Koeberl

Hum Gene Ther· April 2021

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A comprehensive testing algorithm for the diagnosis of Fabry disease in males and females.
Ashlee R Stiles, Haoyue Zhang, Jian Dai, Patricia McCaw, James Beasley, Dwight D Koeberl

Mol Genet Metab· July 2020

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Comparisons of Infant and Adult Mice Reveal Age Effects for Liver Depot Gene Therapy in Pompe Disease.
Sang-Oh Han, Songtao Li, Angela McCall, Benjamin Arnson, Jeffrey I Everitt, Dwight D Koeberl

Mol Ther Methods Clin Dev· June 2020

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Evaluation of antihypertensive drugs in combination with enzyme replacement therapy in mice with Pompe disease.
Sang-Oh Han, Alexina C Haynes, Songtao Li, Dennis M Abraham, Priya S Kishnani, Dwight D Koeberl

Mol Genet Metab· February 2020

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Improved muscle function in a phase I/II clinical trial of albuterol in Pompe disease.
Dwight D Koeberl, Laura E Case, Ankit Desai, Edward C Smith, Crista Walters

Mol Genet Metab· February 2020

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Links between autophagy and disorders of glycogen metabolism - Perspectives on pathogenesis and possible treatments.
Benjamin L Farah, Paul M Yen, Dwight D Koeberl

Mol Genet Metab· January 2020

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Fenofibrate rapidly decreases hepatic lipid and glycogen storage in neonatal mice with glycogen storage disease type Ia.
Zollie A Yavarow, Hye-Ri Kang, Lauren R Waskowicz, Boon-Huat Bay, Sarah P Young, Dwight D Koeberl

Hum Mol Genet· January 2020

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Pathogenesis of Hepatic Tumors following Gene Therapy in Murine and Canine Models of Glycogen Storage Disease.
Hye-Ri Kang, Monika Gjorgjieva, Stephanie N Smith, Elizabeth D Brooks, Zelin Chen, Dwight D Koeberl

Mol Ther Methods Clin Dev· December 2019

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Liver depot gene therapy for Pompe disease.
Priya S Kishnani, Dwight D Koeberl

Ann Transl Med· July 2019

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