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Amelioration of the dystrophic phenotype of mdx mice using a truncated utrophin transgene
- Source :
- Nature. 384:349-353
- Publication Year :
- 1996
- Publisher :
- Springer Science and Business Media LLC, 1996.
-
Abstract
- Duchenne muscular dystrophy (DMD) is a severe, progressive muscle-wasting disease that causes cardiac or respiratory failure and results in death at about 20 years of age. Replacement of the missing protein, dystrophin, using myoblast transfer in humans or viral/liposomal delivery in the mouse DMD model is inefficient and short-lived. One alternative approach to treatment would be to upregulate the closely related protein, utrophin, which might be able to compensate for the dystrophin deficiency in all relevant muscles. As a first step to this approach, we have expressed a utrophin transgene at high levels in the dystrophin-deficient mdx mouse. Our results indicate that high expression of the utrophin transgene in skeletal and diaphragm muscle can markedly reduce the dystrophic pathology. These data suggest that systemic upregulation of utrophin in DMD patients may lead to the development of an effective treatment for this devastating disorder.
- Subjects :
- Male
musculoskeletal diseases
congenital, hereditary, and neonatal diseases and abnormalities
mdx mouse
Utrophin
animal diseases
Transgene
Duchenne muscular dystrophy
Mice, Transgenic
Muscular Dystrophies
Sarcospan
Dystrophin
Mice
Downregulation and upregulation
medicine
Animals
Humans
Myocyte
Transgenes
Creatine Kinase
Genetics
Multidisciplinary
biology
Muscles
Membrane Proteins
Genetic Therapy
musculoskeletal system
medicine.disease
Mice, Mutant Strains
Up-Regulation
Mice, Inbred C57BL
Cytoskeletal Proteins
Gene Expression Regulation
Mice, Inbred CBA
biology.protein
Cancer research
Female
Subjects
Details
- ISSN :
- 14764687 and 00280836
- Volume :
- 384
- Database :
- OpenAIRE
- Journal :
- Nature
- Accession number :
- edsair.doi.dedup.....c7d7717cb90715b477f95361858932fe