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

@article{Lesbordes2003TherapeuticBO,
  title={Therapeutic benefits of cardiotrophin-1 gene transfer in a mouse model of spinal muscular atrophy.},
  author={J C Lesbordes and Carmen Cifuentes-Diaz and Audrey Miroglio and Vandana Joshi and Thierry Bordet and Axel Kahn and Judith Melki},
  journal={Human molecular genetics},
  year={2003},
  volume={12 11},
  pages={1233-9}
}
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 features, similar to those found in human SMA, strongly suggest the involvement of a… CONTINUE READING
27 Citations
28 References
Similar Papers

Citations

Publications citing this paper.
Showing 1-10 of 27 extracted citations

References

Publications referenced by this paper.
Showing 1-10 of 28 references

Similar Papers

Loading similar papers…