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Disease models

Mouse model of amyotrophic lateral sclerosis (ALS) caused by partial loss of TAR DNA binding protein 43 (Tdp-43; Tardbp) function

Mice with partial loss of Tdp-43 function could be useful as models to study and screen for new compounds to treat ALS. TDP-43 aggregation is associated with ALS. Mice were engineered to express artificial microRNA to knock down expression of Tdp-43. The engineered mice recapitulated ALS-like pathophysiology including progressive neuromuscular impairments that eventually led to paralysis and death, and neurodegeneration in the forebrain and spinal cord. Next steps could include using the mouse model to evaluate therapeutic candidates for ALS.

SciBX 7(14); doi:10.1038/scibx.2014.412
Published online April 10, 2014

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Yang, C. et al. Proc. Natl. Acad. Sci. USA; published online
March 10, 2014;
Contact: Zuoshang Xu, University of Massachusetts Medical School, Worcester, Mass.