D. Duan

发表

Parent Project Muscular Dystrophy Females with Dystrophinopathy Conference, Orlando, Florida June 26 - June 27, 2019.

S. Nelson, B. Wong, K. Flanigan, 2020, Journal of neuromuscular diseases.

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

Dongsheng Duan, Feng Zhang, Charles A. Gersbach, 2016, Science.

The evolution of heart gene delivery vectors

D. Duan, N. Wasala, Jin-Hong Shin, 2011, The journal of gene medicine.

Enhancement of Muscle Gene Delivery with Pseudotyped Adeno-Associated Virus Type 5 Correlates with Myoblast Differentiation

D. Duan, J. Engelhardt, Ziying Yan, 2001, Journal of Virology.

Dynamin Is Required for Recombinant Adeno-Associated Virus Type 2 Infection

J. Pessin, A. Kao, D. Duan, 1999, Journal of Virology.

CRISPR-Cas9 cleavage efficiency correlates strongly with target-sgRNA folding stability: from physical mechanism to off-target assessment

Xiaojun Xu, Shi-jie Chen, D. Duan, 2017, Scientific Reports.

Preservation of muscle force in Mdx3cv mice correlates with low-level expression of a near full-length dystrophin protein.

D. Duan, Y. Yue, Dejia Li, 2008, The American journal of pathology.

High-resolution 3D tractography of fibrous tissue based on polarization-sensitive optical coherence tomography

Gang Yao, Dongsheng Duan, D. Duan, 2019, Experimental biology and medicine.

Mapping 3D fiber orientation in tissue using dual-angle optical polarization tractography.

K. Zhang, Y. Wang, D. Duan, 2016, Biomedical optics express.

Considerations on Preclinical Neuromuscular Disease Gene Therapy Studies

D. Duan, 2019, Muscle Gene Therapy.

Muscle Gene Therapy

J. Mendell, D. Duan, 2019, Springer International Publishing.

High resolution imaging of the fibrous microstructure in bovine common carotid artery using optical polarization tractography

Gang Yao, Mohammadreza Ravanfar, Leila Azinfar, 2017, Journal of biophotonics.

Automatic quantification of microscopic heart damage in a mouse model of Duchenne muscular dystrophy using optical polarization tractography

D. Duan, Yuanbo Wang, G. Yao, 2018, Journal of biophotonics.

Heart structural remodeling in a mouse model of Duchenne cardiomyopathy revealed using optical polarization tractography [Invited].

K. Zhang, Y. Wang, D. Duan, 2017, Biomedical optics express.

Late-life restoration of mitochondrial function reverses cardiac dysfunction in old mice

Gennifer E. Merrihew, Ying S. Ting, Lindsay K. Pino, 2020, bioRxiv.

Late-life restoration of mitochondrial function reverses cardiac dysfunction in old mice

Gennifer E. Merrihew, Ying S. Ting, Lindsay K. Pino, 2020, bioRxiv.

Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy.

D. Duan, J. Engelhardt, Ziying Yan, 2000, Proceedings of the National Academy of Sciences of the United States of America.

A new dual-vector approach to enhance recombinant adeno-associated virus-mediated gene expression through intermolecular cis activation

D. Duan, J. Engelhardt, Ziying Yan, 2000, Nature Medicine.

Contemporary Cardiac Issues in Duchenne Muscular Dystrophy

E. Hoffman, D. Judge, G. Pearson, 2015, Circulation.

Ectopic Catalase Expression in Mitochondria by Adeno-Associated Virus Enhances Exercise Performance in Mice

Chady H Hakim, P. Rabinovitch, D. Duan, 2009, PloS one.

Reducing sarcolipin expression mitigates Duchenne muscular dystrophy and associated cardiomyopathy in mice

J. McArdle, J. Mendell, Lai-Hua Xie, 2017, Nature Communications.

α2 and α3 helices of dystrophin R16 and R17 frame a microdomain in the α1 helix of dystrophin R17 for neuronal NOS binding

D. Duan, Y. Yue, Y. Lai, 2012, Proceedings of the National Academy of Sciences.

Circular Intermediates of Recombinant Adeno-Associated Virus Have Defined Structural Characteristics Responsible for Long-Term Episomal Persistence in Muscle Tissue

Prerna Sharma, D. Duan, J. Engelhardt, 1998, Journal of Virology.

A hybrid vector system expands adeno-associated viral vector packaging capacity in a transgene-independent manner.

D. Duan, Arkasubhra Ghosh, Y. Yue, 2008, Molecular therapy : the journal of the American Society of Gene Therapy.

Systemic delivery of adeno-associated viral vectors.

D. Duan, 2016, Current opinion in virology.

Dual AAV Gene Therapy for Duchenne Muscular Dystrophy with a 7-kb Mini-Dystrophin Gene in the Canine Model.

Chady H Hakim, D. Duan, Xiufang Pan, 2017, Human gene therapy.

Duchenne muscular dystrophy animal models for high-throughput drug discovery and precision medicine

Shi-jie Chen, D. Duan, N. Wasala, 2020, Expert opinion on drug discovery.

Questions Answered and Unanswered by the First CRISPR Editing Study in a Canine Model of Duchenne Muscular Dystrophy.

Chady H Hakim, Shi-jie Chen, D. Duan, 2019, Human gene therapy.

Variable rescue of microtubule and physiological phenotypes in mdx muscle expressing different miniaturized dystrophins

J. Ervasti, D. Duan, J. Chamberlain, 2018, Human molecular genetics.

Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy

D. Duan, 2018, Molecular therapy : the journal of the American Society of Gene Therapy.

Delivering Large Therapeutic Genes for Muscle Gene Therapy

D. Duan, Y. Yue, B. Bostick, 2010 .

Adeno-Associated Virus

D. Duan, J. Engelhardt, Y. Yue, 2002 .

Nanotherapy for Duchenne muscular dystrophy.

Chady H Hakim, D. Duan, Chady H. Hakim, 2018, Wiley interdisciplinary reviews. Nanomedicine and nanobiotechnology.

Treating Duchenne Cardiomyopathy in the Mouse Model by Gene Repair

C. Gersbach, D. Duan, N. Wasala, 2017 .

100-fold but not 50-fold dystrophin overexpression aggravates electrocardiographic defects in the mdx model of Duchenne muscular dystrophy

D. Duan, N. Wasala, Y. Yue, 2016, Molecular therapy. Methods & clinical development.

Prospect of gene therapy for cardiomyopathy in hereditary muscular dystrophy

D. Duan, Y. Yue, Timothy L Domeier, 2016, Expert opinion on orphan drugs.

AAV-8 is more efficient than AAV-9 in transducing neonatal dog heart.

Chady H Hakim, D. Duan, Xiufang Pan, 2015, Human gene therapy methods.

Systemic gene transfer reveals distinctive muscle transduction profile of tyrosine mutant AAV-1, -6, and -9 in neonatal dogs

Chady H Hakim, D. Duan, Jin-Hong Shin, 2014, Molecular therapy. Methods & clinical development.

Microdystrophin ameliorates muscular dystrophy in the canine model of duchenne muscular dystrophy.

Chady H Hakim, D. Duan, Xiufang Pan, 2013, Molecular therapy : the journal of the American Society of Gene Therapy.

Humoral immunity to AAV-6, 8, and 9 in normal and dystrophic dogs.

D. Duan, Jin-Hong Shin, Y. Yue, 2012, Human gene therapy.

Progress in gene therapy of dystrophic heart disease

D. Duan, Y. Lai, Yi Lai, 2012, Gene Therapy.

AAV micro-dystrophin gene therapy alleviates stress-induced cardiac death but not myocardial fibrosis in >21-m-old mdx mice, an end-stage model of Duchenne muscular dystrophy cardiomyopathy.

D. Duan, N. Wasala, Jin-Hong Shin, 2012, Journal of molecular and cellular cardiology.

Duchenne muscular dystrophy gene therapy: Lost in translation?

D. Duan, 2011, Research and reports in biology.

AAV-microdystrophin therapy improves cardiac performance in aged female mdx mice.

D. Duan, Jin-Hong Shin, Y. Yue, 2011, Molecular therapy : the journal of the American Society of Gene Therapy.

Duchenne Cardiomyopathy Gene Therapy

D. Duan, Jin-Hong Shin, Y. Yue, 2010 .

Marginal Level Dystrophin Expression Improves Clinical Outcome in a Strain of Dystrophin/Utrophin Double Knockout Mice

D. Duan, Y. Yue, Dejia Li, 2010, PloS one.

Adeno-associated virus serotype-9 microdystrophin gene therapy ameliorates electrocardiographic abnormalities in mdx mice.

D. Duan, Y. Yue, Dejia Li, 2008, Human gene therapy.

A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skeletal muscle transduction in dogs.

J. Kornegay, D. Duan, Arkasubhra Ghosh, 2008, Molecular therapy : the journal of the American Society of Gene Therapy.

Efficient whole-body transduction with trans-splicing adeno-associated viral vectors.

D. Duan, Arkasubhra Ghosh, Y. Yue, 2007, Molecular therapy : the journal of the American Society of Gene Therapy.

Widespread severe myodegeneration in a compound heterozygote female dog with dystrophin deficiency

Chady H Hakim, D. Duan, S. Korte, 2020, Veterinary medicine and science.

Age-matched comparison reveals early electrocardiography and echocardiography changes in dystrophin-deficient dogs

D. Duan, Jin-Hong Shin, Y. Yue, 2011, Neuromuscular Disorders.

An intronic LINE-1 element insertion in the dystrophin gene aborts dystrophin expression and results in Duchenne-like muscular dystrophy in the corgi breed

J. Kornegay, D. Duan, Jin-Hong Shin, 2010, Laboratory Investigation.

Exclusive skeletal muscle correction does not modulate dystrophic heart disease in the aged mdx model of Duchenne cardiomyopathy.

D. Duan, N. Wasala, Y. Yue, 2013, Human molecular genetics.

Dystrophin deficiency impairs vascular structure and function in the canine model of Duchenne muscular dystrophy

D. Duan, M. H. Laughlin, K. Kodippili, 2021, The Journal of pathology.

Adeno-associated virus vectors

D. Duan, J. Engelhardt, 1992 .

Long-term robust myocardial transduction of the dog heart from a peripheral vein by adeno-associated virus serotype-8.

D. Duan, Xiufang Pan, Jin-Hong Shin, 2013, Human gene therapy.

Partial restoration of cardiac function with ΔPDZ nNOS in aged mdx model of Duchenne cardiomyopathy.

D. Duan, N. Wasala, Y. Yue, 2014, Human molecular genetics.

Cardiac-Specific Expression of ΔH2-R15 Mini-Dystrophin Normalized All Electrocardiogram Abnormalities and the End-Diastolic Volume in a 23-Month-Old Mouse Model of Duchenne Dilated Cardiomyopathy.

D. Duan, N. Wasala, Jin-Hong Shin, 2018, Human gene therapy.

Proteomic analysis identifies key differences in the cardiac interactomes of dystrophin and micro-dystrophin

D. Duan, N. Wasala, Y. Yue, 2021, Human molecular genetics.

Challenges and opportunities in dystrophin-deficient cardiomyopathy gene therapy.

D. Duan, 2006, Human molecular genetics.

Sarcolipin overexpression impairs myogenic differentiation in Duchenne muscular dystrophy.

Lai-Hua Xie, D. Duan, N. Niranjan, 2019, American journal of physiology. Cell physiology.

Design of Muscle Gene Therapy Expression Cassette

D. Duan, Y. Lai, Yi Lai, 2019, Muscle Gene Therapy.

Adeno-Associated Virus

D. Duan, J. Engelhardt, Y. Yue, 2002 .

A New Kid on the Playground of CRISPR DMD Therapy.

D. Duan, 2017, Human gene therapy. Clinical development.

Systemic AAV-9 transduction in mice is influenced by animal age but not by the route of administration

D. Duan, Y. Yue, B. Bostick, 2007, Gene Therapy.

High prevalence of plasma lipid abnormalities in human and canine Duchenne and Becker muscular dystrophies depicts a new type of primary genetic dyslipidemia.

Chady H Hakim, G. Francis, F. Rossi, 2020, Journal of clinical lipidology.

A marginal level of dystrophin partially ameliorates hindlimb muscle passive mechanical properties in dystrophin‐null mice

Chady H Hakim, D. Duan, Chady H. Hakim, 2012, Muscle & nerve.

Phenotyping cardiac gene therapy in mice.

D. Duan, Y. Yue, B. Bostick, 2011, Methods in molecular biology.

Full-length dystrophin expression in half of the heart cells ameliorates beta-isoproterenol-induced cardiomyopathy in mdx mice.

D. Duan, Y. Yue, J. Skimming, 2004, Human molecular genetics.

Viral serotype and the transgene sequence influence overlapping adeno‐associated viral (AAV) vector‐mediated gene transfer in skeletal muscle

D. Duan, Arkasubhra Ghosh, Y. Yue, 2006, The journal of gene medicine.

Gender differences in contractile and passive properties of mdx extensor digitorum longus muscle

Chady H Hakim, D. Duan, Chady H. Hakim, 2012, Muscle & nerve.

Gender influences cardiac function in the mdx model of duchenne cardiomyopathy

D. Duan, Y. Yue, B. Bostick, 2010, Muscle & nerve.

Prevention of Dystrophin-Deficient Cardiomyopathy in Twenty-One-Month-Old Carrier Mice by Mosaic Dystrophin Expression or Complementary Dystrophin/Utrophin Expression

D. Duan, Y. Yue, B. Bostick, 2008, Circulation research.

An engineered galactosylceramidase construct improves AAV gene therapy for Krabbe disease in twitcher mice.

S. Sands, D. Duan, Xiufang Pan, 2019, Human gene therapy.

Expanding the capacity of AAV vectors

D. Duan, J. Engelhardt, Ziying Yan, 2005 .

Double strand interaction is the predominant pathway for intermolecular recombination of adeno-associated viral genomes.

D. Duan, Y. Yue, 2003, Virology.

Systemic Delivery of Adeno-Associated Viral Vectors in Mice and Dogs.

Chady H Hakim, D. Duan, Y. Yue, 2019, Methods in molecular biology.

Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus.

Chady H Hakim, D. Duan, Xiufang Pan, 2015, Human molecular genetics.

A simplified immune suppression scheme leads to persistent micro-dystrophin expression in Duchenne muscular dystrophy dogs.

D. Duan, A. Srivastava, Jin-Hong Shin, 2012, Human gene therapy.

Whole body skeletal muscle transduction in neonatal dogs with AAV-9.

D. Duan, Jin-Hong Shin, Y. Yue, 2011, Methods in molecular biology.

Efficient Whole-body Transduction with Trans-splicing Adeno-associated Viral Vectors.

D. Duan, Arkasubhra Ghosh, Y. Yue, 2007, Molecular therapy : the journal of the American Society of Gene Therapy.

Consequences of DNA-Dependent Protein Kinase Catalytic Subunit Deficiency on Recombinant Adeno-Associated Virus Genome Circularization and Heterodimerization in Muscle Tissue

D. Duan, J. Engelhardt, Y. Yue, 2003, Journal of Virology.

Alpha 7 integrin preserves the function of the extensor digitorum longus muscle in dystrophin-null mice.

Chady H Hakim, D. Duan, D. Burkin, 2013, Journal of applied physiology.

iNOS Ablation Does Not Improve Specific Force of the Extensor Digitorum Longus Muscle in Dystrophin-Deficient mdx4cv Mice

D. Duan, Jin-Hong Shin, Dejia Li, 2011, PloS one.

Design of AAV Vectors for Delivery of Large or Multiple Transgenes.

D. Duan, Y. Lai, Junling Zhao, 2019, Methods in molecular biology.

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.

G. Cartee, D. Duan, Xiufang Pan, 2021, Diabetes.

Truncated dystrophins reduce muscle stiffness in the extensor digitorum longus muscle of mdx mice.

Chady H Hakim, D. Duan, Chady H. Hakim, 2013, Journal of applied physiology.

The gRNA vector level determines the outcome of systemic AAV CRISPR therapy for Duchenne muscular dystrophy.

Chady H Hakim, Shi-jie Chen, D. Duan, 2022, Human gene therapy.

CRISPR alleviates muscular dystrophy in dogs

D. Duan, 2018, Nature Biomedical Engineering.

Uniform low-level dystrophin expression in the heart partially preserved cardiac function in an aged mouse model of Duchenne cardiomyopathy.

D. Duan, N. Wasala, Y. Yue, 2017, Journal of molecular and cellular cardiology.

Sarcolemmal nNOS anchoring reveals a qualitative difference between dystrophin and utrophin

K. Davies, D. Duan, J. Chamberlain, 2010, Journal of Cell Science.

The FVB Background Does Not Dramatically Alter the Dystrophic Phenotype of Mdx Mice

Chady H Hakim, D. Duan, N. Wasala, 2015, PLoS currents.

SERCA2a gene transfer improves electrocardiographic performance in aged mdx mice

R. Hajjar, D. Duan, Jin-Hong Shin, 2011, Journal of Translational Medicine.

Systemic Trans-splicing adeno-associated viral delivery efficiently transduces the heart of adult mdx mouse, a model for duchenne muscular dystrophy.

D. Duan, Arkasubhra Ghosh, Jin-Hong Shin, 2009, Human gene therapy.

Cardiac expression of a mini-dystrophin that normalizes skeletal muscle force only partially restores heart function in aged Mdx mice.

J. Chen, D. Duan, Y. Yue, 2009, Molecular therapy : the journal of the American Society of Gene Therapy.

Gene Delivery to the Airway

D. Duan, J. Engelhardt, Yulong Zhang, 1999, Current protocols in human genetics.

Monitoring murine skeletal muscle function for muscle gene therapy.

Chady H Hakim, D. Duan, Dejia Li, 2011, Methods in molecular biology.

Sub-physiological sarcoglycan expression contributes to compensatory muscle protection in mdx mice.

D. Duan, Y. Yue, Dejia Li, 2009, Human molecular genetics.

Nitric oxide‐dependent attenuation of noradrenaline‐induced vasoconstriction is impaired in the canine model of Duchenne muscular dystrophy

Chady H Hakim, D. Duan, Xiufang Pan, 2018, The Journal of physiology.

Trans-splicing adeno-associated viral vector-mediated gene therapy is limited by the accumulation of spliced mRNA but not by dual vector coinfection efficiency.

D. Duan, J. Qiu, D. Pintel, 2004, Human gene therapy.

Dual AAV therapy ameliorates exercise-induced muscle injury and functional ischemia in murine models of Duchenne muscular dystrophy.

Chady H Hakim, D. Duan, Y. Yue, 2013, Human molecular genetics.

Novel mini-dystrophin gene dual adeno-associated virus vectors restore neuronal nitric oxide synthase expression at the sarcolemma.

D. Duan, Yadong Zhang, 2012, Human gene therapy.

Genomic removal of a therapeutic mini-dystrophin gene from adult mice elicits a Duchenne muscular dystrophy-like phenotype.

D. Duan, N. Wasala, Jin-Hong Shin, 2016, Human molecular genetics.

Recombinant AAV-mediated gene delivery using dual vector heterodimerization.

D. Duan, J. Engelhardt, Ziying Yan, 2002, Methods in enzymology.

Recombinant adeno-associated viral vector production and purification.

D. Duan, Jin-Hong Shin, Y. Yue, 2012, Methods in molecular biology.

Adeno-associated virus serotype 6 capsid tyrosine-to-phenylalanine mutations improve gene transfer to skeletal muscle.

D. Duan, Xiao Xiao, G. Jayandharan, 2010, Human gene therapy.

Evidence for the Failure of Adeno-associated Virus Serotype 5 to Package a Viral Genome ≥8.2 kb.

D. Duan, Y. Yue, Y. Lai, 2010, Molecular therapy : the journal of the American Society of Gene Therapy.

Dystrophin contains multiple independent membrane-binding domains.

Chady H Hakim, D. Duan, Xiufang Pan, 2016, Human molecular genetics.

Dystrophin R16/17 protein therapy restores sarcolemmal nNOS in trans and improves muscle perfusion and function

D. Duan, Y. Yue, Keqing Zhang, 2019, Molecular medicine.

Manipulation of mtDNA heteroplasmy in all striated muscles of newborn mice by AAV9-mediated delivery of a mitochondria targeted restriction endonuclease

D. Duan, C. Moraes, S. Williams, 2011, Gene Therapy.

Standard Operating Procedures (SOPs) for Evaluating the Heart in Preclinical Studies of Duchenne Muscular Dystrophy

D. Kass, K. Kinnett, D. Duan, 2016, Journal of Cardiovascular Translational Research.

Gene Therapy: Use of Viruses as Vectors

D. Duan, Michael E. Nance, 2014 .

Canine Models of Inherited Musculoskeletal and Neurodegenerative Diseases

D. Duan, T. Taghian, A. Bradbury, 2020, Frontiers in Veterinary Science.

Duchenne muscular dystrophy

E. Mercuri, S. Takeda, N. Goemans, 2021, Nature Reviews Disease Primers.

Duchenne muscular dystrophy gene therapy in the canine model.

Dongsheng Duan, D. Duan, 2015, Human gene therapy. Clinical development.

Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy

Chady H Hakim, D. Duan, M. Mcintosh, 2015, Disease Models & Mechanisms.

Abnormal Calcium Handling in Duchenne Muscular Dystrophy: Mechanisms and Potential Therapies

D. Duan, G. Babu, Satvik Mareedu, 2021, Frontiers in Physiology.

Rational engineering of a functional CpG-free ITR for AAV gene therapy

D. Duan, Xiufang Pan, D. Pintel, 2021, Gene Therapy.

Single SERCA2a Therapy Ameliorated Dilated Cardiomyopathy for 18 Months in a Mouse Model of Duchenne Muscular Dystrophy.

R. Hajjar, D. Duan, N. Wasala, 2020, Molecular therapy : the journal of the American Society of Gene Therapy.

Reducing sarcolipin expression mitigates Duchenne muscular dystrophy and associated cardiomyopathy in mice

J. McArdle, J. Mendell, Lai-Hua Xie, 2017, Nature Communications.

High-Resolution Histological Landscape of AAV DNA Distribution in Cellular Compartments and Tissues following Local and Systemic Injection

D. Duan, Y. Yue, Keqing Zhang, 2020, Molecular therapy. Methods & clinical development.

Unified energetics analysis unravels SpCas9 cleavage activity for optimal gRNA design

Dongsheng Duan, Dong Zhang, Shi-Jie Chen, 2019, Proceedings of the National Academy of Sciences.

AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice.

Chady H Hakim, Shi-jie Chen, C. Gersbach, 2019, Molecular therapy : the journal of the American Society of Gene Therapy.

A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy

Chady H Hakim, D. Duan, Xiufang Pan, 2017, Molecular therapy. Methods & clinical development.

Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models

Chady H Hakim, D. Grimm, Shi-jie Chen, 2021, Nature Communications.

Modular flexibility of dystrophin: Implications for gene therapy of Duchenne muscular dystrophy

Dongsheng Duan, S. Harper, D. Duan, 2002, Nature Medicine.

Dystrophin Gene Replacement and Gene Repair Therapy for Duchenne Muscular Dystrophy in 2016: An Interview.

D. Duan, 2016, Human gene therapy. Clinical development.

A Translational Pathway Toward a Clinical Trial Using the Second-Generation AAV Micro Dystrophin Vector

D. Duan, Y. Yue, Chady H. Hakim, 2015 .

Expanding Adeno-associated Viral Vector Capacity: A Tale of Two Vectors

D. Duan, Arkasubhra Ghosh, 2007, Biotechnology & genetic engineering reviews.

Synthetic intron improves transduction efficiency of trans-splicing adeno-associated viral vectors.

D. Duan, Y. Yue, Y. Lai, 2006, Human gene therapy.

Brief Report: Synthetic Intron Improves Transduction Efficiency ofTrans-Splicing Adeno-associated Viral Vectors

D. Duan, Y. Yue, Y. Lai, 2006 .

Micro-dystrophin AAV Vectors Made by Transient Transfection and Herpesvirus System Are Equally Potent in Treating mdx Mouse Muscle Disease

B. Byrne, D. Duan, Xiufang Pan, 2020, Molecular therapy. Methods & clinical development.

AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice.

Keqing Zhang, Dongsheng Duan, Charles A. Gersbach, 2018, JCI insight.

Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy

T. Conlon, B. Byrne, M. Styner, 2023, Science Translational Medicine.

Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy.

D. Duan, J. Chamberlain, Y. Yue, 2009, The Journal of clinical investigation.

Evaluation of Muscle Function of the Extensor Digitorum Longus Muscle Ex vivo and Tibialis Anterior Muscle In situ in Mice

Dongsheng Duan, D. Duan, N. Wasala, 2013, Journal of visualized experiments : JoVE.

“Of Mice and Measures”: A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic

K. Davies, M. Girgenrath, C. Lutz, 2018, Journal of neuromuscular diseases.

Dwarf Open Reading Frame (DWORF) Gene Therapy Ameliorated Duchenne Muscular Dystrophy Cardiomyopathy in Aged mdx Mice

D. Duan, Xiufang Pan, Y. Yue, 2023, Journal of the American Heart Association.

Dystrophin R16/17-syntrophin PDZ fusion protein restores sarcolemmal nNOSμ

D. Duan, Y. Yue, Keqing Zhang, 2018, Skeletal Muscle.

Nitrosative stress elicited by nNOSµ delocalization inhibits muscle force in dystrophin‐null mice

D. Duan, Y. Yue, Dejia Li, 2011, Journal of Pathology.

Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors

Dongsheng Duan, Yi Lai, D. Duan, 2005, Nature Biotechnology.

C-terminal-truncated microdystrophin recruits dystrobrevin and syntrophin to the dystrophin-associated glycoprotein complex and reduces muscular dystrophy in symptomatic utrophin/dystrophin double-knockout mice.

D. Duan, Y. Yue, Mingju Liu, 2006, Molecular therapy : the journal of the American Society of Gene Therapy.

Muscle Gene Therapy

D. Duan, 2011, Methods in Molecular Biology.

Characterization of 65 Epitope-Specific Dystrophin Monoclonal Antibodies in Canine and Murine Models of Duchenne Muscular Dystrophy by Immunostaining and Western Blot

D. Duan, M. Mcintosh, Jin-Hong Shin, 2014, PloS one.

Genotyping mdx, mdx3cv, and mdx4cv mice by primer competition polymerase chain reaction

D. Duan, Jin-Hong Shin, Chady H. Hakim, 2011, Muscle & nerve.

Evidence for impaired neurovascular transmission in a murine model of Duchenne muscular dystrophy.

D. Duan, P. Bagher, S. Segal, 2011, Journal of applied physiology.

Evidence for impaired neurovascular transmission in a murine model of 1 Duchenne Muscular Dystrophy 2 3 4 5

D. Duan, P. Bagher, S. Segal, 2010 .

Extensor carpi ulnaris muscle shows unexpected slow-to-fast fiber-type switch in Duchenne muscular dystrophy dogs

Gregory J Jenkins, D. Duan, G. Yao, 2021, Disease models & mechanisms.

Adeno-associated virus-mediated microdystrophin expression protects young mdx muscle from contraction-induced injury.

S. Harper, R. Grange, D. Duan, 2005, Molecular therapy : the journal of the American Society of Gene Therapy.

The implication of hinge 1 and hinge 4 in micro-dystrophin gene therapy for Duchenne muscular dystrophy.

D. Duan, N. Wasala, Y. Yue, 2022, Human gene therapy.

Dystrophin gene replacement and gene repair therapy for Duchenne muscular dystrophy in 2016.

D. Duan, 2016, Human gene therapy. Clinical development.

Dystrophin Deficiency Compromises Force Production of the Extensor Carpi Ulnaris Muscle in the Canine Model of Duchenne Muscular Dystrophy

D. Duan, Xiufang Pan, Jin-Hong Shin, 2012, PloS one.

Microdystrophin Expression as a Surrogate Endpoint for Duchenne Muscular Dystrophy Clinical Trials

O. Danos, D. Duan, J. Chamberlain, 2023, Human gene therapy.

The passive mechanical properties of the extensor digitorum longus muscle are compromised in 2- to 20-mo-old mdx mice.

R. Grange, D. Duan, Chady H. Hakim, 2011, Journal of applied physiology.

Design of trans-splicing adeno-associated viral vectors for Duchenne muscular dystrophy gene therapy.

D. Duan, Y. Yue, Dejia Li, 2008, Methods in molecular biology.

Reply to Head

R. Grange, D. Duan, Chady H. Hakim, 2012 .

Micro-utrophin Therapy for Duchenne Muscular Dystrophy.

D. Duan, 2019, Molecular therapy : the journal of the American Society of Gene Therapy.

Full-length dystrophin reconstitution with adeno-associated viral vectors.

D. Duan, Y. Yue, K. Kodippili, 2014, Human gene therapy.

Sensitive and reliable evaluation of single-cut sgRNAs to restore dystrophin by a GFP-reporter assay

A. Atala, Annemieke Aartsma-Rus, D. Duan, 2020, PloS one.

Microdystrophin Gene Therapy of Cardiomyopathy Restores Dystrophin-Glycoprotein Complex and Improves Sarcolemma Integrity in the Mdx Mouse Heart

S. Harper, D. Duan, J. Chamberlain, 2003, Circulation.

Non-invasive evaluation of muscle disease in the canine model of Duchenne muscular dystrophy by electrical impedance myography

S. Rutkove, D. Duan, J. Coates, 2017, PloS one.

Night Activity Reduction is a Signature Physiological Biomarker for Duchenne Muscular Dystrophy Dogs

D. Duan, G. Yao, Chady H. Hakim, 2015, Journal of neuromuscular diseases.

Perspective on Adeno-Associated Virus Capsid Modification for Duchenne Muscular Dystrophy Gene Therapy.

D. Duan, Michael E. Nance, 2015, Human gene therapy.

Structural analysis of adeno-associated virus transduction circular intermediates.

D. Duan, J. Engelhardt, Ziying Yan, 1999, Virology.

Mechanism of recombinant adeno-associated virus transduction

D. Duan, J. Engelhardt, Ziying Yan, 2005 .

An improved method for studying mouse diaphragm function

Gregory J Jenkins, D. Duan, C. Ambrósio, 2019, Scientific Reports.

Immune Responses to Muscle-Directed AAV Gene Transfer in Clinical Studies.

D. Duan, R. Herzog, Sandeep R. P. Kumar, 2023, Human gene therapy.

A Cautiously Optimistic Outlook of a Designer Therapy for 1% of Duchenne Muscular Dystrophy Patients.

D. Duan, 2021, Human gene therapy.

Efficient transgene reconstitution with hybrid dual AAV vectors carrying the minimized bridging sequences.

D. Duan, Arkasubhra Ghosh, Y. Yue, 2011, Human gene therapy.

Laying the foundation for neuromuscular disease gene therapy.

D. Duan, 2020, Human gene therapy.

Adeno-associated virus serotype-9 efficiently transduces the retinal outer plexiform layer

D. Duan, Arkasubhra Ghosh, Y. Yue, 2009, Molecular vision.

Adeno-associated virus serotype-9 mediated retinal outer plexiform layer transduction is mainly through the photoreceptors.

D. Duan, Arkasubhra Ghosh, Y. Yue, 2010, Advances in experimental medicine and biology.

CRISPR-Cas9 cleavage efficiency correlates strongly with target-sgRNA folding stability: from physical mechanism to off-target assessment

Xiaojun Xu, Shi-jie Chen, D. Duan, 2017, Scientific Reports.

Micro-Dystrophin Gene Therapy Goes Systemic in Duchenne Muscular Dystrophy Patients.

D. Duan, 2018, Human gene therapy.

Development of Next-Generation Muscle Gene Therapy AAV Vectors

D. Duan, Michael E. Nance, 2019, Muscle Gene Therapy.

Formation of Adeno-Associated Virus Circular Genomes Is Differentially Regulated by Adenovirus E4 ORF6 and E2a Gene Expression

Rachael A. Lester, Y. Ye, Prerna Sharma, 1999, Journal of Virology.

A One Health overview, facilitating advances in comparative medicine and translational research

Dongsheng Duan, Ubadah Sabbagh, James P. Calvet, 2016, Clinical and Translational Medicine.

Adenovirus‐mediated gene transfer to adult mouse cardiomyocytes is selectively influenced by culture medium

D. Duan, R. Davisson, Ram V. Sharma, 2003, The journal of gene medicine.

Automatic characterization of stride parameters in canines with a single wearable inertial sensor

Gang Yao, Dongsheng Duan, Gregory J Jenkins, 2018, PloS one.

Quantitative Phenotyping of Duchenne Muscular Dystrophy Dogs by Comprehensive Gait Analysis and Overnight Activity Monitoring

Dongsheng Duan, Zhongna Zhou, Zhihai He, 2013, PloS one.

Catalase overexpression does not impair extensor digitorum longus muscle function in normal mice

D. Duan, Y. Yue, Dejia Li, 2007, Muscle & nerve.

Duchenne Muscular Dystrophy Gene Therapy in 2023: Status, Perspective, and Beyond.

D. Duan, 2023, Human gene therapy.

Early loss of ambulation is not a representative clinical feature in Duchenne muscular dystrophy dogs: remarks on the article of Barthélémy et al.

H. Sweeney, D. Duan, C. Ambrósio, 2015, Disease Models & Mechanisms.

Nondestructive imaging of fiber structure in articular cartilage using optical polarization tractography

Gang Yao, Mohammadreza Ravanfar, Dongsheng Duan, 2016, Journal of biomedical optics.

Two independent molecular pathways for recombinant adeno-associated virus genome conversion occur after UV-C and E4orf6 augmentation of transduction.

D. Duan, J. Engelhardt, S. Sanlioglu, 1999, Human gene therapy.

Myodys, a full-length dystrophin plasmid vector for Duchenne and Becker muscular dystrophy gene therapy.

D. Duan, 2008, Current opinion in molecular therapeutics.

Dual vector expansion of the recombinant AAV packaging capacity.

D. Duan, J. Engelhardt, Y. Yue, 2003, Methods in molecular biology.

Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus.

D. Duan, J. Engelhardt, Ziying Yan, 2000, The Journal of clinical investigation.

Duchenne muscular dystrophy gene therapy in the canine model.

D. Duan, 2015, Human gene therapy. Clinical development.

Duchenne muscular dystrophy gene therapy in the canine model.

D. Duan, 2015, Human gene therapy. Clinical development.

Dystrophin R16/17 protein therapy restores sarcolemmal nNOS in trans and improves muscle perfusion and function

D. Duan, Y. Yue, Keqing Zhang, 2019, Molecular Medicine.

Molecular Pathogenesis of Genetic and Inherited Diseases Preservation of Muscle Force in Mdx3cv Mice Correlates with Low-Level Expression of a Near Full-Length Dystrophin Protein

D. Duan, Y. Yue, Dejia Li, 2008 .

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

G. Cartee, D. Duan, Xiufang Pan, 2023, FASEB journal : official publication of the Federation of American Societies for Experimental Biology.

Expanding AAV packaging capacity with trans-splicing or overlapping vectors: a quantitative comparison.

D. Duan, J. Engelhardt, D. Duan, 2001, Molecular therapy : the journal of the American Society of Gene Therapy.

Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy.

D. Duan, J. Chamberlain, Y. Yue, 2009, The Journal of clinical investigation.

Nitrosative stress elicited by nNOSµ delocalization inhibits muscle force in dystrophin‐null mice

D. Duan, Y. Yue, Dejia Li, 2011, The Journal of pathology.

Optical polarization tractography based on polarization-sensitive optical coherence tomography

Gang Yao, Yuanbo Wang, Mohammadreza Ravanfar, 2016, SPIE BiOS.

Life-long outcomes of systemic AAV micro-dystrophin gene therapy in a murine Duchenne muscular dystrophy model.

D. Duan, N. Wasala, Jin-Hong Shin, 2022, Human gene therapy.

AAV micro-dystrophin gene therapy alleviates stress-induced cardiac death but not myocardial fibrosis in >21-m-old mdx mice, an end-stage model of Duchenne muscular dystrophy cardiomyopathy.

D. Duan, N. Wasala, Jin-Hong Shin, 2012, Journal of molecular and cellular cardiology.

Full-length dystrophin reconstitution with adeno-associated viral vectors.

D. Duan, Y. Yue, K. Kodippili, 2014, Human gene therapy.

Evidence for the failure of adeno-associated virus serotype 5 to package a viral genome > or = 8.2 kb.

D. Duan, Y. Yue, Yi Lai, 2010, Molecular therapy : the journal of the American Society of Gene Therapy.

Incorporation of Adeno-Associated Virus in a Calcium Phosphate Coprecipitate Improves Gene Transfer to Airway Epithelia In Vitro and In Vivo

M. Welsh, D. Duan, J. Engelhardt, 2000, Journal of Virology.

Microdystrophin ameliorates muscular dystrophy in the canine model of duchenne muscular dystrophy.

D. Duan, Xiufang Pan, Jin-Hong Shin, 2013, Molecular therapy : the journal of the American Society of Gene Therapy.

Nondestructive imaging of fiber structure in articular cartilage using optical polarization tractography

Ferris M. Pfeiffer, D. Duan, Yuanbo Wang, 2016, Journal of biomedical optics.

Perspectives on best practices for gene therapy programs.

Hao Wang, K. Klinger, B. Byrne, 2015, Human gene therapy.

499. Intravenous Delivery of a Novel Micro-Dystrophin Vector Prevented Muscle Deterioration in Young Adult Canine Duchenne Muscular Dystrophy Dogs

Gregory J Jenkins, D. Duan, Xiufang Pan, 2016 .