792. Canine Mini-Dystrophin Gene Transfer by AAV1 in mdx Mice Ameliorates Dystrophic Pathology and Protects Membrane Integrity
| Autor: | Bing Wang, Tong Zhu, Joe N. Kornegay, Liqiao Zhou, Chunping Qiao, Mengnan Tian, Xiao Xiao, Janet R. Bogan, Juan Li |
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| Rok vydání: | 2005 |
| Předmět: |
musculoskeletal diseases
Pharmacology Golden retriever muscular dystrophy congenital hereditary and neonatal diseases and abnormalities Pathology medicine.medical_specialty mdx mouse business.industry Duchenne muscular dystrophy Muscle disorder medicine.disease Dystrophin gene nervous system diseases Membrane integrity Small animal Drug Discovery Genetics medicine Molecular Medicine business Molecular Biology Pathological |
| Zdroj: | Molecular Therapy. 11:S308 |
| ISSN: | 1525-0016 |
| Popis: | Duchenne muscular dystrophy (DMD) is the most common and lethal genetic muscle disorders, affecting one in 3,500 males. No efficacious treatment is currently available for DMD. While the mdx mouse is the most widely used small animal model for DMD, the large animal model, golden retriever muscular dystrophy (GRMD) dog, displays more similarities to human DMD patients in clinical and pathological characteristics. Therefore, the GRMD dog is considered a clinically more relevant and therapeutically more challenging DMD model. |
| Databáze: | OpenAIRE |
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