Translational Medicine
Researchers from INSERM discuss findings in translational medicine
Published in Gene Therapy Weekly, October 21st, 2010
Scientists discuss in 'A naturally occurring human minidysferlin protein repairs sarcolemmal lesions in a mouse model of dysferlinopathy' new findings in translational medicine. "Dysferlinopathies are autosomal recessive, progressive muscle dystrophies caused by mutations in DYSF, leading to a loss or a severe reduction of dysferlin, a key protein in sarcolemmal repair. Currently, no etiological treatment is available for patients affected with dysferlinopathy," scientists in Marseille, France report.
"As for other muscular dystrophies, gene therapy approaches based on recombinant adeno-associated virus (rAAV) vectors are promising options. However, because dysferlin...
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Source: Gene Therapy Weekly (2010-10-21)
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