Dongsheng Duan

Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy.

Recombinant AAV-mediated gene delivery using dual vector heterodimerization.

Development of multiple cloning site cis-vectors for recombinant adeno-associated virus production.

Trans-splicing vectors expand the packaging limits of adeno-associated virus for gene therapy applications.

Dual vector expansion of the recombinant AAV packaging capacity.

Consequences of DNA-dependent protein kinase catalytic subunit deficiency on recombinant adeno-associated virus genome circularization and heterodimerization in muscle tissue.

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

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

Microdystrophin gene therapy of cardiomyopathy restores dystrophin-glycoprotein complex and improves sarcolemma integrity in the mdx mouse heart.

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

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

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

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

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

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.

Challenges and opportunities in dystrophin-deficient cardiomyopathy gene therapy.

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

From the smallest virus to the biggest gene: marching towards gene therapy for duchenne muscular dystrophy.

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

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

Prevention of dystrophin-deficient cardiomyopathy in twenty-one-month-old carrier mice by mosaic dystrophin expression or complementary dystrophin/utrophin expression.

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

Expanding adeno-associated viral vector capacity: a tale of two vectors.

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

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

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

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

Dystrophin knockdown mice suggest that early, transient dystrophin expression might be enough to prevent later pathology.

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

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

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

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

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

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

Ectopic catalase expression in mitochondria by adeno-associated virus enhances exercise performance in mice.

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

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

Sarcolemmal nNOS anchoring reveals a qualitative difference between dystrophin and utrophin.

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

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

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

Gender influences cardiac function in the mdx model of Duchenne cardiomyopathy.

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

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

Marginal level dystrophin expression improves clinical outcome in a strain of dystrophin/utrophin double knockout mice.

Monitoring murine skeletal muscle function for muscle gene therapy.

Phenotyping cardiac gene therapy in mice.

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

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

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

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

Duchenne muscular dystrophy gene therapy: Lost in translation?

iNOS ablation does not improve specific force of the extensor digitorum longus muscle in dystrophin-deficient mdx4cv mice.

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

SERCA2a gene transfer improves electrocardiographic performance in aged mdx mice.

The evolution of heart gene delivery vectors.

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

A Simplified Immune Suppression Scheme Leads to Persistent Micro-dystrophin Expression in Duchenne Muscular Dystrophy Dogs.

Gene delivery to the heart: an updated review on vectors and methods.

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

Recombinant adeno-associated viral vector production and purification.

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

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.

Dystrophin deficiency compromises force production of the extensor carpi ulnaris muscle in the canine model of Duchenne muscular dystrophy.

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

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

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

Microdystrophin Ameliorates Muscular Dystrophy in the Canine Model of Duchenne Muscular Dystrophy.