rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice
| Autor: | Jeffrey S. Chamberlain, Paul Gregorevic, James M. Allen, Miki Haraguchi, Elina Minami, Eric E. Finn, Michael J. Blankinship, Marvin E. Adams, Leonard Meuse, Stanley C. Froehner, Charles E. Murry |
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| Rok vydání: | 2006 |
| Předmět: |
musculoskeletal diseases
medicine.medical_specialty Sarcolemma viruses Duchenne muscular dystrophy Genetic enhancement Skeletal muscle General Medicine Anatomy Biology medicine.disease General Biochemistry Genetics and Molecular Biology medicine.anatomical_structure Endocrinology Internal medicine Utrophin medicine biology.protein Respiratory system Muscular dystrophy Dystrophin |
| Zdroj: | Nature Medicine. 12:787-789 |
| ISSN: | 1546-170X 1078-8956 |
| DOI: | 10.1038/nm1439 |
| Popis: | Mice carrying mutations in both the dystrophin and utrophin genes die prematurely as a consequence of severe muscular dystrophy. Here, we show that intravascular administration of recombinant adeno-associated viral (rAAV) vectors carrying a microdystrophin gene restores expression of dystrophin in the respiratory, cardiac and limb musculature of these mice, considerably reducing skeletal muscle pathology and extending lifespan. These findings suggest rAAV vector-mediated systemic gene transfer may be useful for treatment of serious neuromuscular disorders such as Duchenne muscular dystrophy. |
| Databáze: | OpenAIRE |
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