Profile
University of Missouri (MU) medical researcher Dr. Dongsheng Duan is improving the future of young adults with muscular disease.
Duan’s major research interest is in understanding and treating Duchenne muscular dystrophy (DMD), a devastating disease usually leading to death in early adulthood. His work on the topic, which has spanned many years, has focused on the molecular and cellular biology of the disease gene, the disease mechanisms, and possible genetic therapies.
Since he arrived at MU in 2002, Duan has secured more than $30 million in research funding, mainly from the National Institutes of Health. For his many achievements, Duan has received numerous recognitions including the American Society of Gene Therapy’s Outstanding New Investigator award, Spurgeon Distinguished Medical Research Award and Chancellor’s Award for Outstanding Research and Creative Activity from the University of Missouri. Dr. Duan is an elected fellow of the National Academy of Inventors, and an elected fellow of the American Association for the Advancement of Science.
Dr. Dongsheng Duan is a Curators Distinguish Professor and Margaret Proctor Mulligan Professor in Medical Research at the School of Medicine with a joint appointment in the Department of Biomedical Science at the College of Veterinary Medicine, and a joint appointment in the Department of Chemical and Biomedical Engineering at the College of Engineering.
See books and journals that have featured Dr. Duan's Lab on the cover
Academic Information
Margaret Proctor Mulligan Professor in Medical Research Professor
Curators Distinguished Professor
Office
7 Hospital Dr
M658 Medical Sciences Building
Columbia, MO 65212
United States
P. 573-884-9584
Research Interests
- Recombinant adeno-associated virus (rAAV) transduction biology
- Duchenne muscular dystrophy (DMD) gene therapy
- Duchenne cardiomyopathy
- Canine model of muscular dystrophy
- Free radical in muscle function and aging
Areas of Expertise
- Virology and Molecular Therapies
- Gene Therapy
- Neuromuscular biology
- Virology
Related Links
Education & Training
Post-Graduate School
1997, PhD, University of Pennsylvania
Awards & Honors
Committees and Boards
- 2021-Present: Associate Editor “Molecular Therapy”
- 2020 - Present: Associate Editor “Frontiers in Genome Editing
- 2018 – Present: Associate Editor “Human Gene Therapy”
- 2014 – Present: Member, Solid GT Scientific Advisory Committee
- 2014 – Present: Member, Parent Project Muscular Dystrophy (PPMD) Scientific Advisory Committee
- 2011 - Present: Associate Editor “Frontiers in Physiology”
- 2009 – Present: Editorial board “Molecular Therapy”
- 2008 – Present: Editorial board “Gene Therapy”
- 2011 – 2018: Editorial board “PLoS Currents: Muscular Dystrophy”
- 2008 – 2012: NIH SMEP Study Section charter member
- 2010 – 2016: Member, Muscular Dystrophy Association (MDA) Scientific Advisory Committee
Editor
- 2019: Book co-editor for “Muscle Gene Therapy” 2nd edition (Springer, New York, NY)
- 2011: Book editor for “Muscle Gene Therapy: Methods and Protocols” (Humana Press, New York, NY)
- 2010: Book editor for “Muscle Gene Therapy” (Springer, New York, NY)
Awards
- 2021: Curators’ Distinguished Professor
- 2020: Fellow, American Association for the Advancement of Science (AAAS)
- 2019: Fellow, National Academy of Inventors
- 2017: Excellence in Research Mentoring of Trainees Award, University of Missouri, School of Medicine
- 2017: Honorary Medical Alumni Award, University of Missouri
- 2009: Chancellor’s Award for Outstanding Research and Creative Activity, University of Missouri
- 2008: Margaret Proctor Mulligan Professor, University of Missouri
- 2006: Outstanding New Investigator Award, American Society of Gene Therapy
- 2004: Spurgeon Distinguished Medical Research Award, University of Missouri
Publications
2025
- Burke MJ, Blatt BM, Teixeira JA, Perez-Lopez DO, Yue Y, Pan X, Hakim CH, Yao G, Herzog RW, Duan D. Adeno-associated virus-8/9 myofiber type/size tropism profiling reveals therapeutic effect of microdystrophin in canines. Journal of Cachexia, Sarcopenia and Muscle 16(1):e13681, 2025.
- Pérez-López DO, Burke MJ, Hakim CH, Teixeira JA, Han J, Yue Y, Ren Z, Sun J, Chen S-J, Herzog RW, Yao G, Duan D. Circulatory CCL2 distinguishes Duchenne muscular dystrophy dogs. Disease Models and Mechanisms 18(3): dmm052137 2025.
2024
- Li N, Kumar SRP, Cao D, Munoz-Melero M, Sreevani A, Brian BA, Greenwood CM, Yamada K, Duan D, Herzog RW. Redundancy in innate immune pathways that promotes CD8+ T cells responses in AAV1 muscle gene transfer. Viruses 16:1507 2024.
- Morales ED (equal contribution), Wang D (equal contribution), Burke MJ (equal contribution), Han J, Devine DD, Zhang K, Duan D. Transcriptional changes of genes encoding sarcoplasmic reticulum calcium binding and up-taking proteins in normal and Duchenne muscular dystrophy dogs. BMC Musculoskeletal Disorders 16:1507 2024.
- Hu B, Herzog R. Duan D. R-loops facilitate AAV-mediated nuclease-free gene targeting. Molecular Therapy 32(12):4172-4174, 2024.
- Kwak SE, Wang H, Pan X, Duan D, Cartee GD. Genetic reduction of skeletal muscle glycogen synthase 1 abundance reveals that the refeeding-induced reversal of elevated insulin-stimulated glucose uptake after exercise is not attributable to achieving a high muscle glycogen concentration. FASEB Journal 38(22):e70176, 2024.
- Duan D. Full-length dystrophin gene therapy for Duchenne muscular dystrophy. Molecular Therapy 2024 Aug 12:S1525-0016(24)00483-0
- Kwak SE, Zheng A, Arias EB, Wang H, Pan X, Yue Y, Duan D, Cartee GD. A novel genetic model provides a unique perspective on the relationship between postexercise glycogen concentration and increases in the abundance of key metabolic proteins after acute exercise. PLoS ONE 19(1): e0295964, 2024.
- Rana J, Herzog RW, Yamada K, Kumar SRP, Munoz-Melero M, Lam AK, Marusic DM, Duan D, Terhorst C, Byrne BJ, Corti M, Biswas M. B Cell Focused Transient 1 Immune Suppression Protocol for Efficient AAV Readministration to the Liver. Molecular Therapy-Methods & Clinical Development 32(1):101216, 2024.
- Wang H, Kwak SE, Zheng A, Arias EB, Pan X, Duan D, Cartee GD. Phosphorylation of AS160-Serine 704 is not essential for the exercise-induced increase in insulin-stimulated glucose uptake by skeletal muscles from female or male rats. American Journal of Physiology-Endocrinology and Metabolism 326(6):E807-E818, 2024.
- Duan D. Dystrophin and associated proteins. Encyclopedia of the Neurological Sciences. (Third edition). Elsevier. Online April 09, 2024.
- Cao D, Byrne BJ, de Long YP, Terhorst C, Duan D, Herzog RW, Sandeep RP. Innate immune sensing of AAV vectors. Human Gene Therapy 35(13-14):451-463, 2024
- Kodippili K, Hakim HH, Burke MJ, Yue Y, Teixeira JA, Zhang K, Yao G, Babu GJ, Herzog RW, Duan D. SERCA2a overexpression improves muscle function in the canine Duchenne muscular dystrophy model. Molecular Therapy-Methods & Clinical Development 32(2):101268, 2024.
- Wang D, Duan D. Humoral and cellular immune responses to AAV delivery in the airway. Molecular Therapy-Methods & Clinical Development 32(3):101274, 2024.
2023
- Hakim CH, Perez-Lopez D, Burke M, Teixeira J, Duan D.
Molecular and biochemical assessment of gene therapy in the canine model of Duchenne muscular dystrophy.
Methods in Molecular Biology 2587:255-301, 2023 - Hakim CH, Burke M, Teixeira J, Duan D.
Histological assessment of gene therapy in the canine DMD model.
Methods in Molecular Biology 2587:303-338, 2023 - Hakim CH, Teixeira J, Leach SB, Duan D.
Physiological assessment of muscle, heart, and whole-body function in the canine model of Duchenne muscular dystrophy.
Methods in Molecular Biology 2587:67-103, 2023 - Kumar SRP, Duan D, Herzog RW.
Immune Responses to Muscle-Directed AAV Gene Transfer in Clinical Studies.
Human Gene Therapy 34(9-10):365-371, 2023 - Duan D.
Duchenne muscular dystrophy gene therapy in 2023: Status, perspective, and beyond.
Human Gene Therapy 34(9-10):345-349, 2023 - Wang H, Zheng A, Arias EB, Kwak SE, Pan X, Duan D, Cartee GD.
AS160 Expression, but not AS160 Serine-588, Threonine-642, and Serine-704 Phosphorylation, is Essential for Elevated Insulin-stimulated Glucose Uptake by Skeletal Muscle from Female Rats after Acute Exercise.
FASEB Journal 37(7):e23021, 2023. - Duan D.
Lethal immunotoxicity in high-dose systemic AAV gene therapy.
Molecular Therapy 31(11):3123-3126, 2023. - Onyali V, Nourian Z, Boerman EM, Hanft LM, Krenz M, Baines CP, Duan D, McDonald KS, Domeier TL.
Calcium handling dysfunction and cardiac damage following acute ventricular preload challenge in a mouse model of Duchenne muscular dystrophy.
American Journal of Physiology-Heart and Circulatory Physiology
325(5):H1168-H1177, 2023 - Shoti J, Qing K, Keeler GD, Duan D, Byrne BJ, Srivastava A.
Development of capsid- and genome-modified optimized AAVrh74 vectors for muscle gene therapy.
Molecular Therapy-Methods & Clinical Development 31(4):101147, 2023. - Morales ED, Yue Y, Watkins TB, Han J, Pan X, Gibson AM, HB, B-E O, Yao G, Makarewich CA, Babu GJ, Duan D.
Dwarf open reading frame (DWORF) gene therapy ameliorated Duchenne muscular dystrophy cardiomyopathy in aged mdx mice.
Journal of American Heart Association (JAHA) 12:e027480, 2023. - Chamberlain JS, Robb M, Braun S, Brown KJ, Danos O, Ganot A, Gonzalez-Alegre P, Hunter N, McDonald C, Morris C, Tobolowsky M, Wagner KR, Ziolkowski O, Duan D.
Micro-dystrophin expression as a surrogate endpoint for Duchenne muscular dystrophy clinical trials.
Human Gene Therapy 34(9-10):404-415, 2023. - Wasala LP, Watkin TB, Wasala NB, Burke MJ, Yue Y, Lai Y, Yao G, Duan D.
The implication of hinge 1 and hinge 4 in micro-dystrophin gene therapy for Duchenne muscular dystrophy.
Human Gene Therapy 34(9-10):459-470, 2023 - Wasala NB, Yue Y, Hu B, Shin J-H, Yao G, Duan D.
Life-long outcomes of systemic AAV micro-dystrophin gene therapy in a murine Duchenne muscular dystrophy model.
Human Gene Therapy 34(9-10):449-458, 2023 - Birch SM, Law MW, Conlon TJ, Guo L-J, Crudele JM, Hawkins EC, Nghiem PP, Ahn M, Meng H, Beatka MJ, Fickau BA, Prieto JC, Styner MA, Struharik MJ, Shanks C, Brown KJ, Golebiowski D, Bettis AK, Balog-Alvarez CJ, Clement N, Coleman KE, Corti M, Pan X, Hauschka SD, Gonzalez JP, Morris CA, Schneider JS, Duan D, Chamberlain JS, Byrne BJ, Kornegay JN.
A blinded, placebo-controlled systemic gene therapy efficacy study in the GRMD model of Duchenne muscular dystrophy.
Science Translational Medicine 15(677): eabo1815 2023. - Zalcman AR, Hakim CH, Lattimer J, Holland JR, Dodam JR, Duan D.
MRI evaluation of gene therapy in the canine model of Duchenne muscular dystrophy.
Methods in Molecular Biology 2587:339-352, 2023. - Hakim CH, Sandeep RP, Perez-Lopez D, Teixeira J, Herzog RW, Duan D.
Assessment of the gene therapy immune response in the canine muscular dystrophy models.
Methods in Molecular Biology 2587:353-375, 2023.
2022
- Ebner J, Pan, X, Yue Y, Sideromenos S, Koenig X, Hilber K, Duan D.
Na current rescue in dystrophic cardiac Purkinje fibers by micro-dystrophin.
Circulation: Arrhythmia and Electrophysiology 15(8): e011161, 2022. PMID: 35917466; PMCID: PMC9396648; DOI: 10.1161/CIRCEP.122.011161. - Pan X, Yue Y, Boftis M, Wasala LP, Tran NT, Zhang K, Pintel DJ, Tai PWL, Duan D.
Rational engineering of a functional CpG-free ITR for AAV gene therapy.
Gene Therapy, 29(6):333-345, 2022. doi: 10.1038/s41434-021-00296-0 - Duan D*, Flanigan KM, Aartsma-Rus A.
Regarding the article, “Therapeutic Exon Skipping via a CRISPR-guided Cytidine Deaminase Rescues Dystrophic Cardiomyopathy In Vivo”
Circulation 145(18):e872-e873, 2022 (*, corresponding author) - Wasala NB, Million ED, Watkins T, Wasala LP, Han J, Yue Y, Lu B, Chen SJ, Hakim CH, Duan D.
The gRNA vector level determines the outcome of systemic AAV CRISPR therapy for Duchenne muscular dystrophy.
Human Gene Therapy 33(9-10):518-528,, 2022. PMID: 35350865; DOI: 10.1089/hum.2021.130 - Zhang X, Jenkins JG, Hakim CH, Duan D*, Yao G*.
Four-limb wireless IMU sensor system for automatic gait detection in canines (*, co-corresponding author)
Scientific Reports 12:4788, 2022. - Zheng A, Arias EB, Wang H, Kwak SE, Pan X, Duan D, Cartee GD.
The exercise-induced improvement in insulin-stimulated glucose uptake by rat skeletal muscle is absent in male AS160-knockout rats, partially restored by muscle expression of phosphomutated AS160, and fully restored by muscle expression of wildtype AS160.
Diabetes 72(2):219-232, 2022.
2021
- Hakim CH, Kumar SRP, Perez-Lopez DO, Wasala NB, Zhang D, Yue Y, Teixeira J, Pan X, Zhang K, Million ED, Nelson CE, Metzger S, Han J, Louderman JA, Schmidt F, Feng F, Grimm D, Smith BF, Yao G, Yang NN, Gersbach CA, Chen S-J, Herzog RW, Duan D.
Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models.
Nature Communications 12(1),6769, 2021. (Commented in Molecular Therapy 30(1):10-122, 2022, https://pubmed.ncbi.nlm.nih.gov/34895501/; Commented in Human Gene Therapy 32(23-24):1430-1432, 2021. https://pubmed.ncbi.nlm.nih.gov/34935453/; Commented in Gene Therapy online ahead of print 2022. https://pubmed.ncbi.nlm.nih.gov/35194186/ - Hakim CH, Yang HT, Burke MJ, Teixeira J, Jenkins GJ, Yang NN, Yao G, Duan D.
Contractile kinetic analysis reveals unexpected slow to fast myofiber type conversion in the extensor carpi ulnaris muscle of the canine DMD model.
Disease Models and Mechanisms 14(12):dmm049006, 2021. - Kodippili K, Thorne PK, Laughlin MH, Duan D.
Dystrophin deficiency impairs vascular structure and function in the canine model of Duchenne muscular dystrophy.
Journal of Pathology 254(5):589-605, 2021. - Duan D.
A cautiously optimistic outlook of a designer therapy for 1% Duchenne muscular dystrophy patients. Human Gene Therapy 32(17-18):872-874, 2021 - Duan D, Goemans N, Takeda S, Mercuri Eugenio, Aartsma-Rus A.
Duchenne muscular dystrophy.
Nature Reviews Disease Primers 7(1):13, 2021. PMID: 33602943, DOI: 10.1038/s41572-021-00248-3 - Mareedu S, Million ED, Duan D, Babu GJ.
Abnormal Ca++ handling in Duchenne muscular dystrophy: mechanisms and potential therapies.
Frontiers in Physiology 12:647010, 2021. PMID: 33897454, PMCID: PMC8063049, DOI: 10.3389/fphys.2021.647010 - Apkon S, Kinnett K, Cripe L, Duan D, Jackson JL, Kornegay JN, Mah ML, Nelson SF, Rao V, Scavina M, Wong BL, Flanigan KM.
Parent Project Muscular Dystrophy: females with dystrophinopathy conference. Orlando Florida, June 26-27, 2019.
Journal of Neuromuscular Diseases 8(2):315-322, 2021. PMID: 33361607, DOI: 10.3233/JND-200555 - Fortin JS*, Hakim CH, Korte S, Johnson GC, Duan D*.
Widespread severe myodegeneration in a compound heterozygote female dog with dystrophin deficiency.
Veterinary Medicine and Science 7(3):654-659, 2021. (*, co-corresponding author) PMID: 33502125, PMCID: PMC8136971, DOI: 10.1002/vms3.433 - Kodippili K, Thorne PK, Laughlin MH, Duan D.
Dystrophin deficiency impairs vascular structure and function in the canine model of Duchenne muscular dystrophy.
Journal of Pathology 254(5):589-605, 2021. PMID: 33999411, DOI: 10.1002/path.5704 - Wang H, Marrosu E, Brayson D, Wasala NB, Johnson EK, Scott CS, Yue Y, Hau K, Trask AJ, Zhang L, Froehner SC, Adams ME, Duan D, Montanaro F.
Biochemical characterization of micro-dystrophin reveals a role for cavins and ERK in DMD cardiomyopathy.
Human Molecular Genetics, 30(4):1321-1336, 2021
2020
- Lyu P, Yoo, KW, Yadav MK, Atala A, Aartsma-Rus A, Putten MV, Duan D, Lu B.
Sensitive and reliable evaluation of single-cut sgRNAs to restore dystrophin by a GFP-reporter assay.
PLoS One15(9):e0239468, 2020. - Zhao J, Yue Y, Patel A, Wasala LP, Karp JF, Zhang K, Duan D*, Lai Y*.
High-Resolution Histological Landscape of AAV DNA Distribution in Cellular Compartments and Tissues following Local and Systemic Injection.
Molecular Therapy-Methods & Clinical Development 18:856-868, 2020. (*, co-corresponding author). - White Z, Hakim CH, Theret M, Yang NN, Francis G, Cox D, Straub V, Rossi F, Duan D*, Panagiotopoulos C, Bernatchez P*.
High prevalence of plasma lipid abnormalities in human and canine Duchenne and Becker Muscular Dystrophies depicts a new type of primary genetic dyslipidemia.
Journal of Clinical Lipidology. 2020 May 29; S1993-2874 (20)30196-3. Doi:10.1016/j.jacl.2020.05.098 Online ahead of print. PMID: 32593511 (*, co-corresponding author) - Duan D.
Laying the foundation for neuromuscular disease gene therapy.
Human Gene Therapy 31(15-16):785-786, 2020. - Hakim CH, Clement N, Wasala LP, Yang HT, Yue Y, Zhang K, Kodippili K, Adamson-Small L, Pan X, Schneider JS, Yang NN, Chamberlain JS, Byrne BJ, Duan D.
Micro-dystrophin AAV vectors generated by transient transfection and herpesvirus system are similarly effective in protecting muscle disease in dystrophic mice.
Molecular Therapy-Methods & Clinical Development 18:664-678, 2020. PMCID: PMC7403893 PMID: 32775499 - Nance ME, Ravanfar M, Messler M, Duan D*, Yao G*.
PSOCT imaging of muscle degeneration and regeneration in a canine muscle xenograft model.
Biomedical Optics Express 2020 Apr 6;11(5):2383-2393. doi: 10.1364/BOE.390936. eCollection 2020 May 1.PMID: 32499931 (*, co-corresponding author) - Wasala NB, Chen S-J, Duan D.
Duchenne muscular dystrophy animal models for high-throughput drug discovery and precision medicine.
Expert Opinion in Drug Discovery 15(4):443-456, 2020. - Story BD, Miller ME, Bradbury AM, Million ED, Duan D, Taghian T, Fernau D, Beecy SJ, Gray-Edwards HL.
Canine models of inherited musculoskeletal and neurodegenerative diseases.
Frontiers in Veterinary Science 7:80, 2020. https://doi.org/10.3389/fvets.2020.00080 - Yao G, Duan D.
High-resolution 3D tractography of fibrous tissue based on polarization-sensitive optical coherence tomography.
Experimental Biology and Medicine 245(4):273-281, 2020. - Chiao YA, Zhang H, Sweetwyne M, Whitson J, Ting YS, Basisty N, Pino L, Quarles E, Nguyen NT, Campbell M, Zhang T, Gaffrey MJ, Merrihew G, Wang L, Yue Y, Duan D, Granzier H, Szeto HH, Qian W-J, Marcinek D, MacCoss MJ, Rabinovitch PS.
Late-life restoration of mitochondrial function reverses cardiac dysfunction in old mice.
Elife 9:e55513, 2020.
2019
- Wasala NB, Yue Y, Lostal W, Wasala LP, Niranjan N, Hajjar RJ, Babu G, Duan D.
Single SERCA2a Therapy Ameliorated Dilated Cardiomyopathy for 18 Months in a Mouse Model of Duchenne Muscular Dystrophy.
Mol Ther. 2020 Mar 4;28(3):845-854, doi: 10.1016/j.ymthe.2019.12.011. Epub 2020 Jan 10.
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