Development of therapies for spinal muscular atrophy using gene therapy and nanotechnology
Spinal muscular atrophy (SMA) is a genetic disease which is characterized by muscle weakness and atrophy. The disease arises from mutations in the survival motor neuron 1 (SMN1) gene causing degeneration of spinal cord motor neurons. No effective treatment is currently available for SMA however it m...
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University of Sheffield
2013
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| Online Access: | http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.581632 |