Successful regional delivery and long-term expression of a dystrophin gene in canine muscular dystrophy: a preclinical model for human therapies. - Wikidata - awgldk - TriplyDB

Successful regional delivery and long-term expression of a dystrophin gene in canine muscular dystrophy: a preclinical model for human therapies.

Successful regional delivery and long-term expression of a dystrophin gene in canine muscular dystrophy: a preclinical model for human therapies.

http://www.wikidata.org/entity/Q36142496

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The emerging role of viral vectors as vehicles for DMD gene editing Intra-amniotic rAAV-mediated microdystrophin gene transfer improves canine X-linked muscular dystrophy and may induce immune tolerance Advances in gene therapy for muscular dystrophies Dystrophin-deficient cardiomyocytes derived from human urine: new biologic reagents for drug discovery.MicroRNA-29 overexpression by adeno-associated virus suppresses fibrosis and restores muscle function in combination with micro-dystrophin Gene and cell-mediated therapies for muscular dystrophy.The potential of adeno-associated viral vectors for gene delivery to muscle tissue.Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy.Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions.Conserved regions of the DMD 3' UTR regulate translation and mRNA abundance in cultured myotubes Gene therapy for inherited muscle diseases: where genetics meets rehabilitation medicine.Progress and prospects of gene therapy clinical trials for the muscular dystrophies.Phosphorylation within the cysteine-rich region of dystrophin enhances its association with β-dystroglycan and identifies a potential novel therapeutic target for skeletal muscle wasting.In vitro stability of therapeutically relevant, internally truncated dystrophins.Treatment of multifocal breast cancer by systemic delivery of dual-targeted adeno-associated viral vectors.Robust Lentiviral Gene Delivery But Limited Transduction Capacity of Commonly Used Adeno-Associated Viral Serotypes in Xenotransplanted Human Skin.Gene Therapy for Duchenne muscular dystrophy.Microdystrophin ameliorates muscular dystrophy in the canine model of duchenne muscular dystrophy The pharmacology of regenerative medicine Immune responses to AAV vectors: overcoming barriers to successful gene therapy Current Challenges and Future Directions in Recombinant AAV-Mediated Gene Therapy of Duchenne Muscular Dystrophy.Identification of serum protein biomarkers for utrophin based DMD therapy.Gene transfer of arginine kinase to skeletal muscle using adeno-associated virus.Elimination of contaminating cap genes in AAV vector virions reduces immune responses and improves transgene expression in a canine gene therapy model.Repair or replace? Exploiting novel gene and cell therapy strategies for muscular dystrophies.Co-delivery of indoleamine 2,3-dioxygenase prevents loss of expression of an antigenic transgene in dystrophic mouse muscles.Manufacturing of recombinant adeno-associated viruses using mammalian expression platforms.Progress toward Gene Therapy for Duchenne Muscular Dystrophy.AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy.Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy.Regulatory and Exhausted T Cell Responses to AAV Capsid.Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles.Second-generation compound for the modulation of utrophin in the therapy of DMD.Targeted Gene Addition of Microdystrophin in Mice Skeletal Muscle via Human Myoblast Transplantation.Ex vivo intracoronary gene transfer of adeno-associated virus 2 leads to superior transduction over serotypes 8 and 9 in rat heart transplants.Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches.Inhibition of antigen presentation during AAV gene therapy using virus peptides.Multiple Species Comparison of Cardiac Troponin T and Dystrophin: Unravelling the DNA behind Dilated Cardiomyopathy.At the Crossroads of Clinical and Preclinical Research for Muscular Dystrophy-Are We Closer to Effective Treatment for Patients?

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Successful regional delivery and long-term expression of a dystrophin gene in canine muscular dystrophy: a preclinical model for human therapies.

http://www.wikidata.org/entity/Q36142496

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Molecular Therapy

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A Dusty Miller

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P2860

Membrane organization of the dystrophin-glycoprotein complex

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

The polyproline site in hinge 2 influences the functional capacity of truncated dystrophins.

Transduction efficiency and immune response associated with the administration of AAV8 vector into dog skeletal muscle.

Immune responses to AAV in canine muscle monitored by cellular assays and noninvasive imaging.

Expression of human alpha1-antitrypsin in mice and dogs following AAV6 vector-mediated gene transfer to the lungs.

Widespread muscle expression of an AAV9 human mini-dystrophin vector after intravenous injection in neonatal dystrophin-deficient dogs.

Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D.

Dystrophin and mutations: one gene, several proteins, multiple phenotypes.

The dystrophin glycoprotein complex: signaling strength and integrity for the sarcolemma.

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1501-1507

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10.1038/MT.2012.111

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