Targeted gene therapy beneficial to mice with spinal muscular atrophy

Spinal muscular atrophy (SMA) is a neuromuscular disease characterised by progressive muscle wasting and weakness. The severity of the disease varies between individuals, with the clinical spectrum ranging from early infant death to normal adult life with only mild weakness. Currently, there are no effective therapies. As SMA is caused by mutations in the SMN1 gene that result in a lack of SMN protein, gene therapy provides one possible treatment strategy...

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