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Prize4Life and the Jackson Laboratory team up to fight ALS
Date:12/10/2009

ismutase-1 (SOD1) gene. Most ALS research today concentrates on the pathogenesis of SOD1 mutations in laboratory mice. The most widely used ALS mouse models, popularly known as SOD1 mice, are distributed from dedicated supply colonies maintained by The Jackson Laboratory's JAX Breeding Services.

The Laboratory currently distributes 12 different SOD1 models -- with different forms of the SOD1 mutation and on different genetic backgrounds. Among the most widely used of these models is JAX Mice strain B6SJL-Tg(SOD1*G93A)1Gur/J (002726). Like several other SOD1 models, this one has a high copy number of the mutant human superoxide dismutase 1 (SOD1) transgene, which contains the Gly93-->Ala (G93A) substitution. The mutation underlies the most studied form of inherited ALS in humans. The mice lose motor neurons in the spinal cord, become paralyzed in one or more limbs, and die by four to five months. These phenotypes closely model those of human ALS (Gurney et al. 1994).

"An effective treatment for ALS is desperately needed, and the existing [SOD1] mouse model is the primary gateway to clinical trials," wrote prominent ALS researcher Tom Maniatis, Ph.D., on CheckOrphan.org. Maniatis is chair of Columbia University's Biochemistry and Molecular Biophysics Program, a member of Prize4Life's scientific advisory board, and a trustee of The Jackson Laboratory.

Many of the initial studies conducted with Tg(SOD1*G93A)1Gur/J mice have provided a wealth of information and insight on how to best use the mice in preclinical trials. However, like other highly expressed transgenes, the G93A transgene can spontaneously lose copy number, which can greatly confound experimental results. Therefore, the mice need to be handled carefully. When Prize4Life approached The Jackson Laboratory to establish a dedicated supply for their researchers, Chief Operating Officer and Chief Scientific Officer Melanie Leitner, Ph.D. and Scientific Program Officer A. Sheila Menzi
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Contact: Joyce Peterson
joyce.peterson@jax.org
207-288-6058
Jackson Laboratory
Source:Eurekalert

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