University of Missouri medical researcher Dr. Dongsheng Duan is improving the future for 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 mechanism and possible genetic therapies for the disease. In an exciting development, Duan was part of a team that identified a key genetic sequence which is instrumental in DMD. Using their findings, the scientists successfully treated dogs with the disease and are working toward a treatment for humans.
In addition to his medical investigations, Duan’s work includes inspiring future scientists. He invites high school students into his lab to give them hands-on experience conducting research. “We need to give kids an opportunity to see what real science is,” he says.
Since his arrival at MU in 2002, Duan has secured more than $22 million in research funding, mostly 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.
Dr. Dongsheng Duan is Margaret Proctor Mulligan Professor in Medical Research at the School of Medicine with a joint appointment in biomedical sciences at the College of Veterinary Medicine.
M610G Medical Sciences Building
Columbia, MO 65212
- 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
Education & Training
1997, PhD, University of Pennsylvania
Awards & Honors
Committees and Boards
- 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
- 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)
- 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
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. https://pubmed.ncbi.nlm.nih.gov/32970732/
- 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). https://pubmed.ncbi.nlm.nih.gov/32953935/
- 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.
- 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.
- Duan D, Mendell JR. Muscle Gene Therapy (2nd edition). Springer, New York, NY, 2019.
- Hakim CH, Lessa TB, Jenkins GJ, Yang NN, Ambrosio CE, Duan D. An improved method for studying mouse diaphragm function. Scientific Report, 9:19453, 2019.
- Nance ME, Shi R, Hakim CH, Wasala NB, Yue Y, Pan X, Zhang T, Robinson CA, Duan SX, Yao G, Yang NN, Chen S-J, Wagner KR, Gersbach CA, and Duan D. AAV9 edits muscle stem cells in normal and dystrophic adult mice. Molecular Therapy 27(9):1568-1585, 2019
- Pan X, Sands S, Yue Y, Zheng K, LeVine SM, Duan D. An engineered galactosylceramidase construct improves AAV gene therapy for Krabbe disease in twitcher mice. Human Gene Therapy 30(9):1039-51, 2019.
- Zhao J, Yang HT, Wasala LP, Zhang K, Yue Y, Duan D*, Lai Y*. Dystrophin R16/17 protein therapy restores sarcolemmal nNOS in trans and improves muscle perfusion and function. Molecular Medicine 25(1):31, 2019 (*, co-corresponding author)
- Niranjan N, Mareedu S, Tian Y, Kodippili K, Fefelova N, Voit A, Xie L-H, Duan D, Babu GJ. Sarcolipin overexpression impairs myogenic differentiation in Duchenne muscular dystrophy. American Journal of Physiology-Cell Physiology 317(4):C813-C824, 2019.
Zhang D, Hurst T, Duan D, Chen S-J. Unified energetics analysis unravels SpCas9 cleavage activity for optimal gRNA design. Proceedings of National Academy of Science, 116(18):8693-8698, 2019.
Wasala NB, Hakim CH, Chen S-J, Yang NN, Duan D. Questions answered and unanswered by the first CRISPR editing study in the canine model of Duchenne muscular dystrophy. Human Gene Therapy, 30(5):535-543, 2019.
Patel A, Zhao J, Duan D, Lai Y. Design of AAV vectors for Delivery of Large or Multiple Transgenes. Methods in Molecular Biology, 1950:19-33, 2019.
Nance ME, Duan D. Development of next generation muscle gene therapy vectors. Muscle Gene Therapy 2nd edition (Publisher: Springer.) Duan D and Mendel JR (Ed.), 2019. p193-206.
Duan D. Considerations on preclinical muscle gene therapy studies. Muscle Gene Therapy 2nd edition (Publisher: Springer.) Duan D and Mendel JR (Ed.), 2019. p291-326.
Lai Y, Duan D. Design of muscle gene therapy expression cassette. Muscle Gene Therapy 2nd edition (Publisher: Springer.) Duan D and Mendel JR (Ed.), 2019. p141-156.
Wasala LP, Hakim CH, Yue Y, Yang NN, Duan D. Systemic delivery of adeno-associated viral vectors in mice and dogs Methods in Molecular Biology 1937:281-294, 2019.
Hakim CH, Wasala NB, Nelson CE, Wasala LP, Yue Y, Louderman JA, Lessa TB, Zhang K, Jenkins GJ, Nance ME, Pan X, Kodippili K, Yang NN, Chen S-J, Gersbach CA, Duan D. AAV CRISPR editing rescues cardiac and muscle function for 18 months. JCI Insight 3(23): e124297, 2018.
Duan D. CRISPR alleviates muscular dystrophy in dogs. Nature Biomedical Engineering, 2(11):795-796, 2018
Duan D. Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy. Molecular Therapy 26(10):2337-2356, 2018
Duan D. Micro-dystrophin gene therapy goes systemic in Duchenne muscular dystrophy patients. Human Gene Therapy 29(7):733-736, 2018
Kodippili K, Hakim CH, Yang HT, Pan X, Yang NN, Laughlin MH, Terjung RL, Duan D. Nitric oxide dependent attenuation of norepinephrine-induced vasoconstriction is impaired in the canine model of Duchenne muscular dystrophy. Journal of Physiology 596(21):5199-5216
Kodippili K, Duan D. Expressing full-length dystrophin using adeno-associated virus. Gene Therapy in Neurological Disorders Elsevier (Publisher) Ed: Mingjie Li and Joy Snider Chapter 13, 259-276, 2018
Jenkins JG, Hakim CH, Yang NN, Yao G, Duan D. Automatic characterization of stride parameters in canines with a single wearable inertial sensor. PLoS One 13(6): e0198893, 2018.
Wasala NB, Shin J-H, Lai Y, Yue Y, Duan D. Cardiac specific expression of ∆H2-R15 mini-dystrophin normalized all ECG abnormalities and the end-diastolic volume in a 23-m-old mouse model of Duchenne dilated cardiomyopathy. Human Gene Therapy 29(7):737-748, 2018
Patel A, Zhao J, Yue Y, Zhang K, Duan D*, Lai Y*. Dystrophin R16/17-syntrophin PDZ fusion protein restores sarcolemmal nNOS. Skeletal Muscle 8:36, 2018. https://doi.org/10.1186/s13395-018-0182-x (*, co-corresponding author).
Nelson DM, Lindsay A, Judge LM, Duan D, Chamberlain JS, Lowe DA, Ervasti JM. Variable rescue of microtubule and physiological phenotypes in mdx muscle expressing different miniaturized dystrophins. Human Molecular Genetics 27(12):2090-2100, 2018.
Nance ME, Duan D. Gene therapy: use of viruses as vectors. Reference Module in Biomedical Sciences Elsevier. 10-Apr-2018 doi:10.1016/B978-0-12-801238-3.95711-8, 2018. https://doi.org/10.1016/B978-0-12-801238-3.95711-8
Wang Y, Ravanfar M, Zhang K, Duan D, Yao G. Automatic quantification of microscopic heart damage in a mouse model of Duchenne muscular dystrophy using optical polarization tractography. Journal of Biophotonics 11(4):e201700284, 2018.
Gordish-Dressman H, Willmann R, Dalle Pazze L, Kreibich A, van Putten M, Heydemann A, Bogdanik L, Lutz C, Davies K, Demonbruen AR, Duan D, Elsey D, Fukada SI, Girgenrath M, Patrick Gonzalez J, Grounds MD, Nichols A, Partridge T, Passini M, Sanarica F, Schnell FJ, Wells DJ, Yokota T, Young CS, Zhong Z, Spurney C, Spencer M, De Luca A, Nagaraju K, Aartsma-Rus A. "Of Mice and Measures": A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic (First Workshop Report: Examining current findings and opportunities around the emerging D2.B10-Dmdmdx/J (D2/mdx) model in context of the classic C57BL/10ScSn-Dmdmdx/J (Bl10/mdx)). Journal of Neuromuscular Diseases 5(4):407-417, 2018.
Kodippili K, Pan X, Yang HT, Hakim CH, Yue Y, Zhang Y, Shin J-H, Yang NN, Duan D. Duan AAV gene therapy for Duchenne muscular dystrophy with a 7-kb mini-dystrophin gene in the canine model. Human Gene Therapy 29(3):299-311, 2018 (Journal cover image)