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Article

Therapeutic benefits of cardiotrophin-1 gene transfer in a mouse model of spinal muscular atrophy

21 May 2003

Abstract excerpt

Spinal muscular atrophy (SMA) is a recessive autosomal disorder characterized by degeneration of lower motor neurons caused by mutations of the survival motor neuron gene (SMN1). No curative treatment is known so far. Mutant mice carrying homozygous deletion of Smn exon 7 directed to neurons display skeletal muscle denervation, moderate loss of motor neuron cell bodies and severe axonal degeneration. These...

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PMID: 12761038
DOI: 10.1093/hmg/ddg143

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