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Gene therapy improves spinal muscular atrophy in mice
Dec 16, 2004, 17:59, Reviewed by: Dr.
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By Pankaj, US correspondent,
Spinal muscular atrophy is one of the most common genetic diseases leading to death in childhood. Characterized by muscle weakness caused by degeneration of motor neurons in the spinal cord and brain stem nuclei, the condition results from mutations or deletion of the SMN1 gene.
In the December 15 issue of the Journal of Clinical Investigation, Mimoun Azzouz and colleagues from Oxford BioMedica report that multiple single injections of a lentivector expressing human SMN into various muscle of mice with spinal muscular atrophy was able to restore SMN to motor neurons, reduce motor neuron death, and increase life expectancy compared to untreated mice. The authors suggest that this approach may have potential as a safe and practical treatment for many of the symptoms of human spinal muscular atrophy.
- December 15 issue of the Journal of Clinical Investigation
TITLE: Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy
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