Restoring Bcl-x(L) levels benefits a mouse model of spinal muscular atrophy

Neurobiol Dis. 2008 Sep;31(3):361-7. doi: 10.1016/j.nbd.2008.05.014. Epub 2008 Jun 3.

Abstract

Currently, no curative treatment is available for spinal muscular atrophy (SMA). Since the degeneration of spinal motor neurons in SMA is mediated by apoptosis, over-expression of an anti-apoptotic factor, Bcl-x(L), may benefit SMA. Here, we crossed a mouse model of SMA with Bcl-x(L) transgenic mice to create SMA/Bcl-x(L) mice. The Bcl-x(L) expression in the spinal neurons of SMA/Bcl-x(L) mice was nearly double that in SMA mice. SMA/Bcl-x(L) mice showed preserved motor function, normalized electrophysiological tests, diminished muscle atrophy, and less motor neuron degeneration. In addition, the life span of SMA/Bcl-x(L) mice was 1.5 times longer than that of SMA mice. Therefore, over-expression of Bcl-x(L) has a potential for amelioration of SMA, and Bcl-x(L) may be another attractive therapeutic target other than survival motor neuron (SMN) protein for use in future drug screening for SMA.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Apoptosis / genetics
  • Cell Survival / genetics
  • Crosses, Genetic
  • Disease Models, Animal
  • Drug Design
  • Drug Evaluation, Preclinical
  • Genetic Therapy / methods
  • Humans
  • Mice
  • Mice, Inbred C57BL
  • Mice, Knockout
  • Mice, Transgenic
  • Motor Neurons / metabolism*
  • Motor Neurons / pathology
  • Muscular Atrophy / genetics
  • Muscular Atrophy / metabolism
  • Muscular Atrophy / therapy
  • Muscular Atrophy, Spinal / genetics
  • Muscular Atrophy, Spinal / metabolism*
  • Muscular Atrophy, Spinal / therapy*
  • Nerve Degeneration / genetics
  • Nerve Degeneration / metabolism
  • Nerve Degeneration / therapy
  • Spinal Cord / metabolism*
  • Spinal Cord / pathology
  • Spinal Cord / physiopathology
  • Survival Rate
  • bcl-X Protein / genetics
  • bcl-X Protein / metabolism*

Substances

  • bcl-X Protein